Zobrazeno 1 - 9
of 9
pro vyhledávání: '"Van Haren KP"'
Autor:
Hashemi E; Department of Neurology and Neurological Sciences, Stanford University School of Medicine, Stanford, CA., Srivastava IN; Department of Neurology and Neurological Sciences, Stanford University School of Medicine, Stanford, CA., Aguirre A; Department of Neurology and Neurological Sciences, Stanford University School of Medicine, Stanford, CA., Yoseph ET; Department of Neurology and Neurological Sciences, Stanford University School of Medicine, Stanford, CA., Kaushal E; Department of Neurology and Neurological Sciences, Stanford University School of Medicine, Stanford, CA., Awani A; Department of Neurology and Neurological Sciences, Stanford University School of Medicine, Stanford, CA., Ryu JK; Gladstone Institute for Neurological Disease, San Francisco, CA.; Center for Neurovascular Brain Immunology at Gladstone and UCSF, San Francisco, CA.; Department of Neurology, Weill Institute for Neurosciences, University of California San Francisco, San Francisco, CA., Akassoglou K; Gladstone Institute for Neurological Disease, San Francisco, CA.; Center for Neurovascular Brain Immunology at Gladstone and UCSF, San Francisco, CA.; Department of Neurology, Weill Institute for Neurosciences, University of California San Francisco, San Francisco, CA., Talebian S; Department of Neurology and Neurological Sciences, Stanford University School of Medicine, Stanford, CA., Chu P; Stanford Human Research Histology Core, Stanford University School of Medicine, Stanford, CA., Pisani L; Department of Radiology, Stanford University School of Medicine Stanford, Stanford, CA., Musolino P; Department of Neurology, Massachusetts General Hospital, Boston, MA.; Center for Genomic Medicine, Massachusetts General Hospital, Boston, MA., Steinman L; Department of Neurology and Neurological Sciences, Stanford University School of Medicine, Stanford, CA., Doyle K; Department of Immunobiology, University of Arizona, Tucson, AZ., Robinson WH; Department of Immunology and Rheumatology, Stanford University School of Medicine, Stanford, CA., Sharpe O; Department of Immunology and Rheumatology, Stanford University School of Medicine, Stanford, CA., Cayrol R; Department of Pathology, Clinical Department of Laboratory Medicine, University of Montreal, Quebec, Canada., Orchard PJ; Division of Pediatric Blood and Marrow Transplantation, University of Minnesota, Minneapolis, MN., Lund T; Division of Pediatric Blood and Marrow Transplantation, University of Minnesota, Minneapolis, MN., Vogel H; Department of Pathology, Stanford University School of Medicine, Stanford, CA., Lenail M; Department of Neurology and Neurological Sciences, Stanford University School of Medicine, Stanford, CA., Han MH; Department of Neurology and Neurological Sciences, Stanford University School of Medicine, Stanford, CA., Bonkowsky JL; Division of Pediatric Neurology, Department of Pediatrics, University of Utah School of Medicine, Salt Lake City, UT.; Brain and Spine Center, Primary Children's Hospital, Salt Lake City, UT.; Primary Children's Center for Personalized Medicine, Salt Lake City, UT., Van Haren KP; Department of Neurology and Neurological Sciences, Stanford University School of Medicine, Stanford, CA.; Department of Pediatrics, Stanford University School of Medicine, Stanford, CA.
Publikováno v:
Annals of neurology [Ann Neurol] 2024 Oct 28. Date of Electronic Publication: 2024 Oct 28.
Autor:
Adang LA; Division of Neurology, Children's Hospital of Philadelphia, Philadelphia, PA, USA. Electronic address: adangl@chop.edu., Sevagamoorthy A; Division of Neurology, Children's Hospital of Philadelphia, Philadelphia, PA, USA., Sherbini O; Division of Neurology, Children's Hospital of Philadelphia, Philadelphia, PA, USA., Fraser JL; Rare Disease Institute, Children's National Medical Center, Washington, DC, USA; Leukodystrophy and Myelin Disorders Program, Children's National Medical Center, Washington, DC, USA., Bonkowsky JL; Division of Pediatric Neurology, Department of Pediatrics, University of Utah School of Medicine, Salt Lake City, UT, USA; Center for Personalized Medicine, Primary Children's Hospital, Salt Lake City, UT, USA., Gavazzi F; Division of Neurology, Children's Hospital of Philadelphia, Philadelphia, PA, USA., D'Aiello R; Division of Neurology, Children's Hospital of Philadelphia, Philadelphia, PA, USA., Modesti NB; Division of Neurology, Children's Hospital of Philadelphia, Philadelphia, PA, USA., Yu E; Division of Neurology, Children's Hospital of Philadelphia, Philadelphia, PA, USA., Mutua S; Division of Neurology, Children's Hospital of Philadelphia, Philadelphia, PA, USA., Kotes E; Division of Neurology, Children's Hospital of Philadelphia, Philadelphia, PA, USA., Shults J; Department of Biomedical and Health Informatics, Children's Hospital of Philadelphia, Philadelphia, PA, USA; Center for Clinical Epidemiology and Biostatistics, University of Pennsylvania, Philadelphia, PA, USA., Vincent A; CHOP Research Institute, Children's Hospital of Philadelphia, Philadelphia, PA, USA., Emrick LT; Division of Neurology and Developmental Neuroscience in Department Pediatrics, Baylor College Medicine and Texas Children's Hospital, Houston, TX, USA; Department of Human and Molecular Genetics, Baylor College of Medicine, Houston, TX, USA., Keller S; Children's Healthcare of Atlanta Scottish Rite Hospital, Emory University School of Medicine, Atlanta, GA, USA., Van Haren KP; Department of Neurology, Stanford University, Stanford, CA, USA., Woidill S; Division of Neurology, Children's Hospital of Philadelphia, Philadelphia, PA, USA., Barcelos I; Division of Neurology, Children's Hospital of Philadelphia, Philadelphia, PA, USA., Pizzino A; Division of Neurology, Children's Hospital of Philadelphia, Philadelphia, PA, USA., Schmidt JL; Division of Neurology, Children's Hospital of Philadelphia, Philadelphia, PA, USA., Eichler F; Department of Neurology, Massachusetts General Hospital, Boston, MA, USA., Fatemi A; Moser Center for Leukodystrophies, Kennedy Krieger Institute, Baltimore, MD, USA; Departments of Neurology & Pediatrics, Johns Hopkins School of Medicine, Baltimore, MD, USA., Vanderver A; Division of Neurology, Children's Hospital of Philadelphia, Philadelphia, PA, USA.
Publikováno v:
Molecular genetics and metabolism [Mol Genet Metab] 2024 May; Vol. 142 (1), pp. 108453. Date of Electronic Publication: 2024 Mar 18.
Autor:
Srivastava IN; From the Department of Neurology, School of Medicine, Stanford University, Palo Alto, CA. i.srivastava@stanford.edu., Van Haren KP; From the Department of Neurology, School of Medicine, Stanford University, Palo Alto, CA.
Publikováno v:
Neurology [Neurology] 2023 Nov 27; Vol. 101 (22), pp. 985-986. Date of Electronic Publication: 2023 Nov 27.
Autor:
Hashemi E; Department of Neurology and Neurological Sciences, Stanford University School of Medicine, Stanford, CA, USA., Narain Srivastava I; Department of Neurology and Neurological Sciences, Stanford University School of Medicine, Stanford, CA, USA., Aguirre A; Department of Neurology and Neurological Sciences, Stanford University School of Medicine, Stanford, CA, USA., Tilahan Yoseph E; Department of Neurology and Neurological Sciences, Stanford University School of Medicine, Stanford, CA, USA., Kaushal E; Department of Neurology and Neurological Sciences, Stanford University School of Medicine, Stanford, CA, USA., Awani A; Department of Neurology and Neurological Sciences, Stanford University School of Medicine, Stanford, CA, USA., Kyu Ryu J; Gladstone Institute for Neurological Disease; San Francisco, CA, USA.; Center for Neurovascular Brain Immunology at Gladstone and UCSF; San Francisco, CA USA.; Department of Neurology, Weill Institute for Neurosciences, University of California San Francisco; San Francisco, CA, USA., Akassoglou K; Gladstone Institute for Neurological Disease; San Francisco, CA, USA.; Center for Neurovascular Brain Immunology at Gladstone and UCSF; San Francisco, CA USA.; Department of Neurology, Weill Institute for Neurosciences, University of California San Francisco; San Francisco, CA, USA., Talebian S; Department of Neurology and Neurological Sciences, Stanford University School of Medicine, Stanford, CA, USA., Chu P; Stanford Human Research Histology Core, Stanford University School of Medicine, Stanford, CA, USA., Pisani L; Department of Radiology, Stanford University School of Medicine Stanford, CA, USA., Musolino P; Department of Neurology, Massachusetts General Hospital, Boston, MA, USA.; Center for Genomic Medicine, Massachusetts General Hospital, Boston, MA, USA., Steinman L; Department of Neurology and Neurological Sciences, Stanford University School of Medicine, Stanford, CA, USA., Doyle K; Department of Immunobiology, University of Arizona, Tucson, AZ, USA., Robinson WH; Department of Immunology & Rheumatology, Stanford University School of Medicine, Stanford, CA, USA., Sharpe O; Department of Immunology & Rheumatology, Stanford University School of Medicine, Stanford, CA, USA., Cayrol R; Department of Pathology, Clinical Department of Laboratory Medicine, University of Montreal, Quebec, Canada., Orchard P; Division of Pediatric Blood & Marrow Transplantation, University of Minnesota, Minneapolis, MN, USA., Lund T; Division of Pediatric Blood & Marrow Transplantation, University of Minnesota, Minneapolis, MN, USA., Vogel H; Departments of Pathology, Stanford University School of Medicine, Stanford, CA, USA., Lenail M; Department of Neurology and Neurological Sciences, Stanford University School of Medicine, Stanford, CA, USA., Han MH; Department of Neurology and Neurological Sciences, Stanford University School of Medicine, Stanford, CA, USA., Bonkowsky JL; Division of Pediatric Neurology, Department of Pediatrics, University of Utah School of Medicine, Salt Lake City, Utah.; Brain and Spine Center, Primary Children's Hospital, Salt Lake City, Utah.; Primary Children's Center for Personalized Medicine, Salt Lake City, Utah., Van Haren KP; Department of Neurology and Neurological Sciences, Stanford University School of Medicine, Stanford, CA, USA.; Department of Pediatrics, Stanford University School of Medicine, Stanford, CA, USA.
Publikováno v:
BioRxiv : the preprint server for biology [bioRxiv] 2023 Nov 10. Date of Electronic Publication: 2023 Nov 10.
Autor:
van Haren KP; Division of Child Neurology, Department of Neurology Stanford University California Palo Alto USA., Wilkes J; Intermountain Healthcare Utah Salt Lake City USA., Moser AB; Peroxisomal Disease Laboratory Kennedy Krieger Institute Maryland Baltimore USA., Raymond GV; Department of Neurology Johns Hopkins University School of Medicine Maryland Baltimore USA.; Department of Genetic Medicine Johns Hopkins University School of Medicine Maryland Baltimore USA., Richardson T; Research and Statistics Children's Hospital Association Kansas Lenexa USA., Aubourg P; INSERM U 1169 Paris-Sud University Le Kremlin-Bicêtre France.; Department of Pediatric Neurology, APHP Bicêtre University Hospital Le Kremlin-Bicêtre France., Collins TW; Department of Geography University of Utah Utah Salt Lake City USA., Mowry EM; Department of Neurology Johns Hopkins University School of Medicine Maryland Baltimore USA., Bonkowsky JL; Division of Pediatric Neurology, Department of Pediatrics University of Utah School of Medicine Utah Salt Lake City USA.; Brain and Spine Center Primary Children's Hospital Utah Salt Lake City USA.; Primary Children's Center for Personalized Medicine Utah Salt Lake City USA.
Publikováno v:
Annals of the Child Neurology Society [Ann Child Neurol Soc] 2023 Jun; Vol. 1 (2), pp. 155-161. Date of Electronic Publication: 2023 Apr 18.
Autor:
Van Haren KP; Department of Neurology (K.P.V.H., A.A., D.P., L.C.C., N.C.R., J.W., L.S.), Department of Pediatrics (K.P.V.H., T.C., G.M.E., L.S.), Quantitative Sciences Unit (K.C., V.S.) and Department of Radiology (M.G., D.S.), Stanford University School of Medicine Palo Alto, CA; Russell H. Morgan Department of Radiology and Radiological Science (M.P., P.B.B.), The Johns Hopkins University School of Medicine; The Kennedy Krieger Institute (M.P., P.B.B., A.F.); Department of Genetic Medicine (G.V.R.), The Johns Hopkins University School of Medicine, Baltimore, MD; Department of Pathology (T.C.), Stanford University School of Medicine, Palo Alto, CA; Department of Neurology (E.W.), University of California at San Francisco, ; and Department of Neurology (A.F.), The Johns Hopkins University School of Medicine, Baltimore, MD., Cunanan K; Department of Neurology (K.P.V.H., A.A., D.P., L.C.C., N.C.R., J.W., L.S.), Department of Pediatrics (K.P.V.H., T.C., G.M.E., L.S.), Quantitative Sciences Unit (K.C., V.S.) and Department of Radiology (M.G., D.S.), Stanford University School of Medicine Palo Alto, CA; Russell H. Morgan Department of Radiology and Radiological Science (M.P., P.B.B.), The Johns Hopkins University School of Medicine; The Kennedy Krieger Institute (M.P., P.B.B., A.F.); Department of Genetic Medicine (G.V.R.), The Johns Hopkins University School of Medicine, Baltimore, MD; Department of Pathology (T.C.), Stanford University School of Medicine, Palo Alto, CA; Department of Neurology (E.W.), University of California at San Francisco, ; and Department of Neurology (A.F.), The Johns Hopkins University School of Medicine, Baltimore, MD., Awani A; Department of Neurology (K.P.V.H., A.A., D.P., L.C.C., N.C.R., J.W., L.S.), Department of Pediatrics (K.P.V.H., T.C., G.M.E., L.S.), Quantitative Sciences Unit (K.C., V.S.) and Department of Radiology (M.G., D.S.), Stanford University School of Medicine Palo Alto, CA; Russell H. Morgan Department of Radiology and Radiological Science (M.P., P.B.B.), The Johns Hopkins University School of Medicine; The Kennedy Krieger Institute (M.P., P.B.B., A.F.); Department of Genetic Medicine (G.V.R.), The Johns Hopkins University School of Medicine, Baltimore, MD; Department of Pathology (T.C.), Stanford University School of Medicine, Palo Alto, CA; Department of Neurology (E.W.), University of California at San Francisco, ; and Department of Neurology (A.F.), The Johns Hopkins University School of Medicine, Baltimore, MD., Gu M; Department of Neurology (K.P.V.H., A.A., D.P., L.C.C., N.C.R., J.W., L.S.), Department of Pediatrics (K.P.V.H., T.C., G.M.E., L.S.), Quantitative Sciences Unit (K.C., V.S.) and Department of Radiology (M.G., D.S.), Stanford University School of Medicine Palo Alto, CA; Russell H. Morgan Department of Radiology and Radiological Science (M.P., P.B.B.), The Johns Hopkins University School of Medicine; The Kennedy Krieger Institute (M.P., P.B.B., A.F.); Department of Genetic Medicine (G.V.R.), The Johns Hopkins University School of Medicine, Baltimore, MD; Department of Pathology (T.C.), Stanford University School of Medicine, Palo Alto, CA; Department of Neurology (E.W.), University of California at San Francisco, ; and Department of Neurology (A.F.), The Johns Hopkins University School of Medicine, Baltimore, MD., Peña D; Department of Neurology (K.P.V.H., A.A., D.P., L.C.C., N.C.R., J.W., L.S.), Department of Pediatrics (K.P.V.H., T.C., G.M.E., L.S.), Quantitative Sciences Unit (K.C., V.S.) and Department of Radiology (M.G., D.S.), Stanford University School of Medicine Palo Alto, CA; Russell H. Morgan Department of Radiology and Radiological Science (M.P., P.B.B.), The Johns Hopkins University School of Medicine; The Kennedy Krieger Institute (M.P., P.B.B., A.F.); Department of Genetic Medicine (G.V.R.), The Johns Hopkins University School of Medicine, Baltimore, MD; Department of Pathology (T.C.), Stanford University School of Medicine, Palo Alto, CA; Department of Neurology (E.W.), University of California at San Francisco, ; and Department of Neurology (A.F.), The Johns Hopkins University School of Medicine, Baltimore, MD., Chromik LC; Department of Neurology (K.P.V.H., A.A., D.P., L.C.C., N.C.R., J.W., L.S.), Department of Pediatrics (K.P.V.H., T.C., G.M.E., L.S.), Quantitative Sciences Unit (K.C., V.S.) and Department of Radiology (M.G., D.S.), Stanford University School of Medicine Palo Alto, CA; Russell H. Morgan Department of Radiology and Radiological Science (M.P., P.B.B.), The Johns Hopkins University School of Medicine; The Kennedy Krieger Institute (M.P., P.B.B., A.F.); Department of Genetic Medicine (G.V.R.), The Johns Hopkins University School of Medicine, Baltimore, MD; Department of Pathology (T.C.), Stanford University School of Medicine, Palo Alto, CA; Department of Neurology (E.W.), University of California at San Francisco, ; and Department of Neurology (A.F.), The Johns Hopkins University School of Medicine, Baltimore, MD., Považan M; Department of Neurology (K.P.V.H., A.A., D.P., L.C.C., N.C.R., J.W., L.S.), Department of Pediatrics (K.P.V.H., T.C., G.M.E., L.S.), Quantitative Sciences Unit (K.C., V.S.) and Department of Radiology (M.G., D.S.), Stanford University School of Medicine Palo Alto, CA; Russell H. Morgan Department of Radiology and Radiological Science (M.P., P.B.B.), The Johns Hopkins University School of Medicine; The Kennedy Krieger Institute (M.P., P.B.B., A.F.); Department of Genetic Medicine (G.V.R.), The Johns Hopkins University School of Medicine, Baltimore, MD; Department of Pathology (T.C.), Stanford University School of Medicine, Palo Alto, CA; Department of Neurology (E.W.), University of California at San Francisco, ; and Department of Neurology (A.F.), The Johns Hopkins University School of Medicine, Baltimore, MD., Rossi NC; Department of Neurology (K.P.V.H., A.A., D.P., L.C.C., N.C.R., J.W., L.S.), Department of Pediatrics (K.P.V.H., T.C., G.M.E., L.S.), Quantitative Sciences Unit (K.C., V.S.) and Department of Radiology (M.G., D.S.), Stanford University School of Medicine Palo Alto, CA; Russell H. Morgan Department of Radiology and Radiological Science (M.P., P.B.B.), The Johns Hopkins University School of Medicine; The Kennedy Krieger Institute (M.P., P.B.B., A.F.); Department of Genetic Medicine (G.V.R.), The Johns Hopkins University School of Medicine, Baltimore, MD; Department of Pathology (T.C.), Stanford University School of Medicine, Palo Alto, CA; Department of Neurology (E.W.), University of California at San Francisco, ; and Department of Neurology (A.F.), The Johns Hopkins University School of Medicine, Baltimore, MD., Goodman J; Department of Neurology (K.P.V.H., A.A., D.P., L.C.C., N.C.R., J.W., L.S.), Department of Pediatrics (K.P.V.H., T.C., G.M.E., L.S.), Quantitative Sciences Unit (K.C., V.S.) and Department of Radiology (M.G., D.S.), Stanford University School of Medicine Palo Alto, CA; Russell H. Morgan Department of Radiology and Radiological Science (M.P., P.B.B.), The Johns Hopkins University School of Medicine; The Kennedy Krieger Institute (M.P., P.B.B., A.F.); Department of Genetic Medicine (G.V.R.), The Johns Hopkins University School of Medicine, Baltimore, MD; Department of Pathology (T.C.), Stanford University School of Medicine, Palo Alto, CA; Department of Neurology (E.W.), University of California at San Francisco, ; and Department of Neurology (A.F.), The Johns Hopkins University School of Medicine, Baltimore, MD., Sundaram V; Department of Neurology (K.P.V.H., A.A., D.P., L.C.C., N.C.R., J.W., L.S.), Department of Pediatrics (K.P.V.H., T.C., G.M.E., L.S.), Quantitative Sciences Unit (K.C., V.S.) and Department of Radiology (M.G., D.S.), Stanford University School of Medicine Palo Alto, CA; Russell H. Morgan Department of Radiology and Radiological Science (M.P., P.B.B.), The Johns Hopkins University School of Medicine; The Kennedy Krieger Institute (M.P., P.B.B., A.F.); Department of Genetic Medicine (G.V.R.), The Johns Hopkins University School of Medicine, Baltimore, MD; Department of Pathology (T.C.), Stanford University School of Medicine, Palo Alto, CA; Department of Neurology (E.W.), University of California at San Francisco, ; and Department of Neurology (A.F.), The Johns Hopkins University School of Medicine, Baltimore, MD., Winterbottom J; Department of Neurology (K.P.V.H., A.A., D.P., L.C.C., N.C.R., J.W., L.S.), Department of Pediatrics (K.P.V.H., T.C., G.M.E., L.S.), Quantitative Sciences Unit (K.C., V.S.) and Department of Radiology (M.G., D.S.), Stanford University School of Medicine Palo Alto, CA; Russell H. Morgan Department of Radiology and Radiological Science (M.P., P.B.B.), The Johns Hopkins University School of Medicine; The Kennedy Krieger Institute (M.P., P.B.B., A.F.); Department of Genetic Medicine (G.V.R.), The Johns Hopkins University School of Medicine, Baltimore, MD; Department of Pathology (T.C.), Stanford University School of Medicine, Palo Alto, CA; Department of Neurology (E.W.), University of California at San Francisco, ; and Department of Neurology (A.F.), The Johns Hopkins University School of Medicine, Baltimore, MD., Raymond GV; Department of Neurology (K.P.V.H., A.A., D.P., L.C.C., N.C.R., J.W., L.S.), Department of Pediatrics (K.P.V.H., T.C., G.M.E., L.S.), Quantitative Sciences Unit (K.C., V.S.) and Department of Radiology (M.G., D.S.), Stanford University School of Medicine Palo Alto, CA; Russell H. Morgan Department of Radiology and Radiological Science (M.P., P.B.B.), The Johns Hopkins University School of Medicine; The Kennedy Krieger Institute (M.P., P.B.B., A.F.); Department of Genetic Medicine (G.V.R.), The Johns Hopkins University School of Medicine, Baltimore, MD; Department of Pathology (T.C.), Stanford University School of Medicine, Palo Alto, CA; Department of Neurology (E.W.), University of California at San Francisco, ; and Department of Neurology (A.F.), The Johns Hopkins University School of Medicine, Baltimore, MD., Cowan T; Department of Neurology (K.P.V.H., A.A., D.P., L.C.C., N.C.R., J.W., L.S.), Department of Pediatrics (K.P.V.H., T.C., G.M.E., L.S.), Quantitative Sciences Unit (K.C., V.S.) and Department of Radiology (M.G., D.S.), Stanford University School of Medicine Palo Alto, CA; Russell H. Morgan Department of Radiology and Radiological Science (M.P., P.B.B.), The Johns Hopkins University School of Medicine; The Kennedy Krieger Institute (M.P., P.B.B., A.F.); Department of Genetic Medicine (G.V.R.), The Johns Hopkins University School of Medicine, Baltimore, MD; Department of Pathology (T.C.), Stanford University School of Medicine, Palo Alto, CA; Department of Neurology (E.W.), University of California at San Francisco, ; and Department of Neurology (A.F.), The Johns Hopkins University School of Medicine, Baltimore, MD., Enns GM; Department of Neurology (K.P.V.H., A.A., D.P., L.C.C., N.C.R., J.W., L.S.), Department of Pediatrics (K.P.V.H., T.C., G.M.E., L.S.), Quantitative Sciences Unit (K.C., V.S.) and Department of Radiology (M.G., D.S.), Stanford University School of Medicine Palo Alto, CA; Russell H. Morgan Department of Radiology and Radiological Science (M.P., P.B.B.), The Johns Hopkins University School of Medicine; The Kennedy Krieger Institute (M.P., P.B.B., A.F.); Department of Genetic Medicine (G.V.R.), The Johns Hopkins University School of Medicine, Baltimore, MD; Department of Pathology (T.C.), Stanford University School of Medicine, Palo Alto, CA; Department of Neurology (E.W.), University of California at San Francisco, ; and Department of Neurology (A.F.), The Johns Hopkins University School of Medicine, Baltimore, MD., Waubant E; Department of Neurology (K.P.V.H., A.A., D.P., L.C.C., N.C.R., J.W., L.S.), Department of Pediatrics (K.P.V.H., T.C., G.M.E., L.S.), Quantitative Sciences Unit (K.C., V.S.) and Department of Radiology (M.G., D.S.), Stanford University School of Medicine Palo Alto, CA; Russell H. Morgan Department of Radiology and Radiological Science (M.P., P.B.B.), The Johns Hopkins University School of Medicine; The Kennedy Krieger Institute (M.P., P.B.B., A.F.); Department of Genetic Medicine (G.V.R.), The Johns Hopkins University School of Medicine, Baltimore, MD; Department of Pathology (T.C.), Stanford University School of Medicine, Palo Alto, CA; Department of Neurology (E.W.), University of California at San Francisco, ; and Department of Neurology (A.F.), The Johns Hopkins University School of Medicine, Baltimore, MD., Steinman L; Department of Neurology (K.P.V.H., A.A., D.P., L.C.C., N.C.R., J.W., L.S.), Department of Pediatrics (K.P.V.H., T.C., G.M.E., L.S.), Quantitative Sciences Unit (K.C., V.S.) and Department of Radiology (M.G., D.S.), Stanford University School of Medicine Palo Alto, CA; Russell H. Morgan Department of Radiology and Radiological Science (M.P., P.B.B.), The Johns Hopkins University School of Medicine; The Kennedy Krieger Institute (M.P., P.B.B., A.F.); Department of Genetic Medicine (G.V.R.), The Johns Hopkins University School of Medicine, Baltimore, MD; Department of Pathology (T.C.), Stanford University School of Medicine, Palo Alto, CA; Department of Neurology (E.W.), University of California at San Francisco, ; and Department of Neurology (A.F.), The Johns Hopkins University School of Medicine, Baltimore, MD., Barker PB; Department of Neurology (K.P.V.H., A.A., D.P., L.C.C., N.C.R., J.W., L.S.), Department of Pediatrics (K.P.V.H., T.C., G.M.E., L.S.), Quantitative Sciences Unit (K.C., V.S.) and Department of Radiology (M.G., D.S.), Stanford University School of Medicine Palo Alto, CA; Russell H. Morgan Department of Radiology and Radiological Science (M.P., P.B.B.), The Johns Hopkins University School of Medicine; The Kennedy Krieger Institute (M.P., P.B.B., A.F.); Department of Genetic Medicine (G.V.R.), The Johns Hopkins University School of Medicine, Baltimore, MD; Department of Pathology (T.C.), Stanford University School of Medicine, Palo Alto, CA; Department of Neurology (E.W.), University of California at San Francisco, ; and Department of Neurology (A.F.), The Johns Hopkins University School of Medicine, Baltimore, MD., Spielman D; Department of Neurology (K.P.V.H., A.A., D.P., L.C.C., N.C.R., J.W., L.S.), Department of Pediatrics (K.P.V.H., T.C., G.M.E., L.S.), Quantitative Sciences Unit (K.C., V.S.) and Department of Radiology (M.G., D.S.), Stanford University School of Medicine Palo Alto, CA; Russell H. Morgan Department of Radiology and Radiological Science (M.P., P.B.B.), The Johns Hopkins University School of Medicine; The Kennedy Krieger Institute (M.P., P.B.B., A.F.); Department of Genetic Medicine (G.V.R.), The Johns Hopkins University School of Medicine, Baltimore, MD; Department of Pathology (T.C.), Stanford University School of Medicine, Palo Alto, CA; Department of Neurology (E.W.), University of California at San Francisco, ; and Department of Neurology (A.F.), The Johns Hopkins University School of Medicine, Baltimore, MD., Fatemi A; Department of Neurology (K.P.V.H., A.A., D.P., L.C.C., N.C.R., J.W., L.S.), Department of Pediatrics (K.P.V.H., T.C., G.M.E., L.S.), Quantitative Sciences Unit (K.C., V.S.) and Department of Radiology (M.G., D.S.), Stanford University School of Medicine Palo Alto, CA; Russell H. Morgan Department of Radiology and Radiological Science (M.P., P.B.B.), The Johns Hopkins University School of Medicine; The Kennedy Krieger Institute (M.P., P.B.B., A.F.); Department of Genetic Medicine (G.V.R.), The Johns Hopkins University School of Medicine, Baltimore, MD; Department of Pathology (T.C.), Stanford University School of Medicine, Palo Alto, CA; Department of Neurology (E.W.), University of California at San Francisco, ; and Department of Neurology (A.F.), The Johns Hopkins University School of Medicine, Baltimore, MD.
Publikováno v:
Neurology. Genetics [Neurol Genet] 2023 Mar 31; Vol. 9 (2), pp. e200061. Date of Electronic Publication: 2023 Mar 31 (Print Publication: 2023).
Autor:
Engelen M; From the Department of Pediatric Neurology/Emma Children's Hospital (M.E., W.J.C.B., I.C.H.), Amsterdam UMC, Amsterdam Leukodystrophy Center, University of Amsterdam, the Netherlands; Division of Child Neurology (E.J.M.), Department of Pediatrics, Weill Cornell Medicine/NewYork-Presbyterian Hospital, NY; Department of Neurology & Pediatrics/Lucile Packard Children's Hospital (K.P.V.H.), Stanford University School of Medicine, Palo Alto, CA 4. Department of Neurology, Leukodystrophy Clinic, University of Leipzig Medical Center, Germany; Unit of Rare Neurodegenerative and Neurometabolic Diseases (E.S.), Fondazione IRCCS Istituto Neurologico C. Besta, Milano, Italy; Department of Pediatric Endocrinology/Emma Children's Hospital (A.S.P.T.), Amsterdam UMC, University of Amsterdam, the Netherlands; AP-HP (F.M.), Department of Medical Genetics, Reference Center for Adult Neurometabolic Diseases and Leukodystrophies, and INSERM U 1127, CNRS UMR 7225, Paris Brain Institute, La Pitié-Salpêtrière University Hospital, Paris, France; Department of Pediatric Neurology/Hôpital Bicêtre Paris Sud (C.S.), France, Reference Center for Children Leukodystrophies Inserm U1127, ICM-Hôpital Pitié Salpêtrière, Paris, France; Division of Pediatric Endocrinology and Diabetes (M.O.R.), Children's Hospital at Montefiore, Albert Einstein College of Medicine of Medicine, Bronx, NY; Neuroendocrine Unit (N.A.T.), Massachusetts General Hospital, Boston, MA; Harvard Medical School (N.A.T.), Boston, MA; Division of Pediatric Endocrinology (A.H.), Department of Pediatrics, Massachusetts General Hospital, Boston, MA, and Harvard Medical School (A.H.), Boston, MA; Charles Dent Metabolic Unit (R.H.L.), National Hospital for Neurology and Neurosurgery, London, United Kingdom; Metabolic Medicine (J.D.), Great Ormond Street Hospital for Children, London United Kingdom; Department of Genetic Medicine (G.V.R.), Johns Hopkins, Baltimore, MD; Division of Pediatric Blood and Marrow Transplantation & Cellular Therapy (T.L., P.J.O.), University of Minnesota, Minneapolis; Pediatric Oncology (J.-S.K.), Hematology, Hemostaseology, University Hospital Leipzig, Germany; Pediatric Blood and Bone Marrow Transplantation (C.A.L.), Princess Maxima Center Utrecht, the Netherlands; Department of Pediatrics (C.A.L.), Wilhemina Children's Hospital, UMC Utrecht, Utrecht University, the Netherlands; Director of Pediatric Neuroimaging (P.C.), Lenox Hill Radiology and Medical Imaging Associates, New York, NY; Moser Center for Leukodystrophies (B.R.T., A.F.), Kennedy Krieger Institute, Johns Hopkins Medical Institutions, Baltimore, MD; Department of Neurogenetics (A.B.M.), Kennedy Krieger Institute, Baltimore, MD; Laboratory Genetic Metabolic Diseases (F.M.V., S.F., S.K.), Department of Clinical Chemistry and Pediatrics, Amsterdam UMC, Amsterdam Gastroenterology Endocrinology Metabolism, University of Amsterdam, the Netherlands; and Department of Neurology (F.S.E.), Massachusetts General Hospital, Boston, MA. Dr. van Ballegoij is currently at the Department of Neurology, Zaans Medisch Centrum, Zaandam. m.engelen@amsterdamumc.nl., van Ballegoij WJC; From the Department of Pediatric Neurology/Emma Children's Hospital (M.E., W.J.C.B., I.C.H.), Amsterdam UMC, Amsterdam Leukodystrophy Center, University of Amsterdam, the Netherlands; Division of Child Neurology (E.J.M.), Department of Pediatrics, Weill Cornell Medicine/NewYork-Presbyterian Hospital, NY; Department of Neurology & Pediatrics/Lucile Packard Children's Hospital (K.P.V.H.), Stanford University School of Medicine, Palo Alto, CA 4. Department of Neurology, Leukodystrophy Clinic, University of Leipzig Medical Center, Germany; Unit of Rare Neurodegenerative and Neurometabolic Diseases (E.S.), Fondazione IRCCS Istituto Neurologico C. Besta, Milano, Italy; Department of Pediatric Endocrinology/Emma Children's Hospital (A.S.P.T.), Amsterdam UMC, University of Amsterdam, the Netherlands; AP-HP (F.M.), Department of Medical Genetics, Reference Center for Adult Neurometabolic Diseases and Leukodystrophies, and INSERM U 1127, CNRS UMR 7225, Paris Brain Institute, La Pitié-Salpêtrière University Hospital, Paris, France; Department of Pediatric Neurology/Hôpital Bicêtre Paris Sud (C.S.), France, Reference Center for Children Leukodystrophies Inserm U1127, ICM-Hôpital Pitié Salpêtrière, Paris, France; Division of Pediatric Endocrinology and Diabetes (M.O.R.), Children's Hospital at Montefiore, Albert Einstein College of Medicine of Medicine, Bronx, NY; Neuroendocrine Unit (N.A.T.), Massachusetts General Hospital, Boston, MA; Harvard Medical School (N.A.T.), Boston, MA; Division of Pediatric Endocrinology (A.H.), Department of Pediatrics, Massachusetts General Hospital, Boston, MA, and Harvard Medical School (A.H.), Boston, MA; Charles Dent Metabolic Unit (R.H.L.), National Hospital for Neurology and Neurosurgery, London, United Kingdom; Metabolic Medicine (J.D.), Great Ormond Street Hospital for Children, London United Kingdom; Department of Genetic Medicine (G.V.R.), Johns Hopkins, Baltimore, MD; Division of Pediatric Blood and Marrow Transplantation & Cellular Therapy (T.L., P.J.O.), University of Minnesota, Minneapolis; Pediatric Oncology (J.-S.K.), Hematology, Hemostaseology, University Hospital Leipzig, Germany; Pediatric Blood and Bone Marrow Transplantation (C.A.L.), Princess Maxima Center Utrecht, the Netherlands; Department of Pediatrics (C.A.L.), Wilhemina Children's Hospital, UMC Utrecht, Utrecht University, the Netherlands; Director of Pediatric Neuroimaging (P.C.), Lenox Hill Radiology and Medical Imaging Associates, New York, NY; Moser Center for Leukodystrophies (B.R.T., A.F.), Kennedy Krieger Institute, Johns Hopkins Medical Institutions, Baltimore, MD; Department of Neurogenetics (A.B.M.), Kennedy Krieger Institute, Baltimore, MD; Laboratory Genetic Metabolic Diseases (F.M.V., S.F., S.K.), Department of Clinical Chemistry and Pediatrics, Amsterdam UMC, Amsterdam Gastroenterology Endocrinology Metabolism, University of Amsterdam, the Netherlands; and Department of Neurology (F.S.E.), Massachusetts General Hospital, Boston, MA. Dr. van Ballegoij is currently at the Department of Neurology, Zaans Medisch Centrum, Zaandam., Mallack EJ; From the Department of Pediatric Neurology/Emma Children's Hospital (M.E., W.J.C.B., I.C.H.), Amsterdam UMC, Amsterdam Leukodystrophy Center, University of Amsterdam, the Netherlands; Division of Child Neurology (E.J.M.), Department of Pediatrics, Weill Cornell Medicine/NewYork-Presbyterian Hospital, NY; Department of Neurology & Pediatrics/Lucile Packard Children's Hospital (K.P.V.H.), Stanford University School of Medicine, Palo Alto, CA 4. Department of Neurology, Leukodystrophy Clinic, University of Leipzig Medical Center, Germany; Unit of Rare Neurodegenerative and Neurometabolic Diseases (E.S.), Fondazione IRCCS Istituto Neurologico C. Besta, Milano, Italy; Department of Pediatric Endocrinology/Emma Children's Hospital (A.S.P.T.), Amsterdam UMC, University of Amsterdam, the Netherlands; AP-HP (F.M.), Department of Medical Genetics, Reference Center for Adult Neurometabolic Diseases and Leukodystrophies, and INSERM U 1127, CNRS UMR 7225, Paris Brain Institute, La Pitié-Salpêtrière University Hospital, Paris, France; Department of Pediatric Neurology/Hôpital Bicêtre Paris Sud (C.S.), France, Reference Center for Children Leukodystrophies Inserm U1127, ICM-Hôpital Pitié Salpêtrière, Paris, France; Division of Pediatric Endocrinology and Diabetes (M.O.R.), Children's Hospital at Montefiore, Albert Einstein College of Medicine of Medicine, Bronx, NY; Neuroendocrine Unit (N.A.T.), Massachusetts General Hospital, Boston, MA; Harvard Medical School (N.A.T.), Boston, MA; Division of Pediatric Endocrinology (A.H.), Department of Pediatrics, Massachusetts General Hospital, Boston, MA, and Harvard Medical School (A.H.), Boston, MA; Charles Dent Metabolic Unit (R.H.L.), National Hospital for Neurology and Neurosurgery, London, United Kingdom; Metabolic Medicine (J.D.), Great Ormond Street Hospital for Children, London United Kingdom; Department of Genetic Medicine (G.V.R.), Johns Hopkins, Baltimore, MD; Division of Pediatric Blood and Marrow Transplantation & Cellular Therapy (T.L., P.J.O.), University of Minnesota, Minneapolis; Pediatric Oncology (J.-S.K.), Hematology, Hemostaseology, University Hospital Leipzig, Germany; Pediatric Blood and Bone Marrow Transplantation (C.A.L.), Princess Maxima Center Utrecht, the Netherlands; Department of Pediatrics (C.A.L.), Wilhemina Children's Hospital, UMC Utrecht, Utrecht University, the Netherlands; Director of Pediatric Neuroimaging (P.C.), Lenox Hill Radiology and Medical Imaging Associates, New York, NY; Moser Center for Leukodystrophies (B.R.T., A.F.), Kennedy Krieger Institute, Johns Hopkins Medical Institutions, Baltimore, MD; Department of Neurogenetics (A.B.M.), Kennedy Krieger Institute, Baltimore, MD; Laboratory Genetic Metabolic Diseases (F.M.V., S.F., S.K.), Department of Clinical Chemistry and Pediatrics, Amsterdam UMC, Amsterdam Gastroenterology Endocrinology Metabolism, University of Amsterdam, the Netherlands; and Department of Neurology (F.S.E.), Massachusetts General Hospital, Boston, MA. Dr. van Ballegoij is currently at the Department of Neurology, Zaans Medisch Centrum, Zaandam., Van Haren KP; From the Department of Pediatric Neurology/Emma Children's Hospital (M.E., W.J.C.B., I.C.H.), Amsterdam UMC, Amsterdam Leukodystrophy Center, University of Amsterdam, the Netherlands; Division of Child Neurology (E.J.M.), Department of Pediatrics, Weill Cornell Medicine/NewYork-Presbyterian Hospital, NY; Department of Neurology & Pediatrics/Lucile Packard Children's Hospital (K.P.V.H.), Stanford University School of Medicine, Palo Alto, CA 4. Department of Neurology, Leukodystrophy Clinic, University of Leipzig Medical Center, Germany; Unit of Rare Neurodegenerative and Neurometabolic Diseases (E.S.), Fondazione IRCCS Istituto Neurologico C. Besta, Milano, Italy; Department of Pediatric Endocrinology/Emma Children's Hospital (A.S.P.T.), Amsterdam UMC, University of Amsterdam, the Netherlands; AP-HP (F.M.), Department of Medical Genetics, Reference Center for Adult Neurometabolic Diseases and Leukodystrophies, and INSERM U 1127, CNRS UMR 7225, Paris Brain Institute, La Pitié-Salpêtrière University Hospital, Paris, France; Department of Pediatric Neurology/Hôpital Bicêtre Paris Sud (C.S.), France, Reference Center for Children Leukodystrophies Inserm U1127, ICM-Hôpital Pitié Salpêtrière, Paris, France; Division of Pediatric Endocrinology and Diabetes (M.O.R.), Children's Hospital at Montefiore, Albert Einstein College of Medicine of Medicine, Bronx, NY; Neuroendocrine Unit (N.A.T.), Massachusetts General Hospital, Boston, MA; Harvard Medical School (N.A.T.), Boston, MA; Division of Pediatric Endocrinology (A.H.), Department of Pediatrics, Massachusetts General Hospital, Boston, MA, and Harvard Medical School (A.H.), Boston, MA; Charles Dent Metabolic Unit (R.H.L.), National Hospital for Neurology and Neurosurgery, London, United Kingdom; Metabolic Medicine (J.D.), Great Ormond Street Hospital for Children, London United Kingdom; Department of Genetic Medicine (G.V.R.), Johns Hopkins, Baltimore, MD; Division of Pediatric Blood and Marrow Transplantation & Cellular Therapy (T.L., P.J.O.), University of Minnesota, Minneapolis; Pediatric Oncology (J.-S.K.), Hematology, Hemostaseology, University Hospital Leipzig, Germany; Pediatric Blood and Bone Marrow Transplantation (C.A.L.), Princess Maxima Center Utrecht, the Netherlands; Department of Pediatrics (C.A.L.), Wilhemina Children's Hospital, UMC Utrecht, Utrecht University, the Netherlands; Director of Pediatric Neuroimaging (P.C.), Lenox Hill Radiology and Medical Imaging Associates, New York, NY; Moser Center for Leukodystrophies (B.R.T., A.F.), Kennedy Krieger Institute, Johns Hopkins Medical Institutions, Baltimore, MD; Department of Neurogenetics (A.B.M.), Kennedy Krieger Institute, Baltimore, MD; Laboratory Genetic Metabolic Diseases (F.M.V., S.F., S.K.), Department of Clinical Chemistry and Pediatrics, Amsterdam UMC, Amsterdam Gastroenterology Endocrinology Metabolism, University of Amsterdam, the Netherlands; and Department of Neurology (F.S.E.), Massachusetts General Hospital, Boston, MA. Dr. van Ballegoij is currently at the Department of Neurology, Zaans Medisch Centrum, Zaandam., Köhler W; From the Department of Pediatric Neurology/Emma Children's Hospital (M.E., W.J.C.B., I.C.H.), Amsterdam UMC, Amsterdam Leukodystrophy Center, University of Amsterdam, the Netherlands; Division of Child Neurology (E.J.M.), Department of Pediatrics, Weill Cornell Medicine/NewYork-Presbyterian Hospital, NY; Department of Neurology & Pediatrics/Lucile Packard Children's Hospital (K.P.V.H.), Stanford University School of Medicine, Palo Alto, CA 4. Department of Neurology, Leukodystrophy Clinic, University of Leipzig Medical Center, Germany; Unit of Rare Neurodegenerative and Neurometabolic Diseases (E.S.), Fondazione IRCCS Istituto Neurologico C. Besta, Milano, Italy; Department of Pediatric Endocrinology/Emma Children's Hospital (A.S.P.T.), Amsterdam UMC, University of Amsterdam, the Netherlands; AP-HP (F.M.), Department of Medical Genetics, Reference Center for Adult Neurometabolic Diseases and Leukodystrophies, and INSERM U 1127, CNRS UMR 7225, Paris Brain Institute, La Pitié-Salpêtrière University Hospital, Paris, France; Department of Pediatric Neurology/Hôpital Bicêtre Paris Sud (C.S.), France, Reference Center for Children Leukodystrophies Inserm U1127, ICM-Hôpital Pitié Salpêtrière, Paris, France; Division of Pediatric Endocrinology and Diabetes (M.O.R.), Children's Hospital at Montefiore, Albert Einstein College of Medicine of Medicine, Bronx, NY; Neuroendocrine Unit (N.A.T.), Massachusetts General Hospital, Boston, MA; Harvard Medical School (N.A.T.), Boston, MA; Division of Pediatric Endocrinology (A.H.), Department of Pediatrics, Massachusetts General Hospital, Boston, MA, and Harvard Medical School (A.H.), Boston, MA; Charles Dent Metabolic Unit (R.H.L.), National Hospital for Neurology and Neurosurgery, London, United Kingdom; Metabolic Medicine (J.D.), Great Ormond Street Hospital for Children, London United Kingdom; Department of Genetic Medicine (G.V.R.), Johns Hopkins, Baltimore, MD; Division of Pediatric Blood and Marrow Transplantation & Cellular Therapy (T.L., P.J.O.), University of Minnesota, Minneapolis; Pediatric Oncology (J.-S.K.), Hematology, Hemostaseology, University Hospital Leipzig, Germany; Pediatric Blood and Bone Marrow Transplantation (C.A.L.), Princess Maxima Center Utrecht, the Netherlands; Department of Pediatrics (C.A.L.), Wilhemina Children's Hospital, UMC Utrecht, Utrecht University, the Netherlands; Director of Pediatric Neuroimaging (P.C.), Lenox Hill Radiology and Medical Imaging Associates, New York, NY; Moser Center for Leukodystrophies (B.R.T., A.F.), Kennedy Krieger Institute, Johns Hopkins Medical Institutions, Baltimore, MD; Department of Neurogenetics (A.B.M.), Kennedy Krieger Institute, Baltimore, MD; Laboratory Genetic Metabolic Diseases (F.M.V., S.F., S.K.), Department of Clinical Chemistry and Pediatrics, Amsterdam UMC, Amsterdam Gastroenterology Endocrinology Metabolism, University of Amsterdam, the Netherlands; and Department of Neurology (F.S.E.), Massachusetts General Hospital, Boston, MA. Dr. van Ballegoij is currently at the Department of Neurology, Zaans Medisch Centrum, Zaandam., Salsano E; From the Department of Pediatric Neurology/Emma Children's Hospital (M.E., W.J.C.B., I.C.H.), Amsterdam UMC, Amsterdam Leukodystrophy Center, University of Amsterdam, the Netherlands; Division of Child Neurology (E.J.M.), Department of Pediatrics, Weill Cornell Medicine/NewYork-Presbyterian Hospital, NY; Department of Neurology & Pediatrics/Lucile Packard Children's Hospital (K.P.V.H.), Stanford University School of Medicine, Palo Alto, CA 4. Department of Neurology, Leukodystrophy Clinic, University of Leipzig Medical Center, Germany; Unit of Rare Neurodegenerative and Neurometabolic Diseases (E.S.), Fondazione IRCCS Istituto Neurologico C. Besta, Milano, Italy; Department of Pediatric Endocrinology/Emma Children's Hospital (A.S.P.T.), Amsterdam UMC, University of Amsterdam, the Netherlands; AP-HP (F.M.), Department of Medical Genetics, Reference Center for Adult Neurometabolic Diseases and Leukodystrophies, and INSERM U 1127, CNRS UMR 7225, Paris Brain Institute, La Pitié-Salpêtrière University Hospital, Paris, France; Department of Pediatric Neurology/Hôpital Bicêtre Paris Sud (C.S.), France, Reference Center for Children Leukodystrophies Inserm U1127, ICM-Hôpital Pitié Salpêtrière, Paris, France; Division of Pediatric Endocrinology and Diabetes (M.O.R.), Children's Hospital at Montefiore, Albert Einstein College of Medicine of Medicine, Bronx, NY; Neuroendocrine Unit (N.A.T.), Massachusetts General Hospital, Boston, MA; Harvard Medical School (N.A.T.), Boston, MA; Division of Pediatric Endocrinology (A.H.), Department of Pediatrics, Massachusetts General Hospital, Boston, MA, and Harvard Medical School (A.H.), Boston, MA; Charles Dent Metabolic Unit (R.H.L.), National Hospital for Neurology and Neurosurgery, London, United Kingdom; Metabolic Medicine (J.D.), Great Ormond Street Hospital for Children, London United Kingdom; Department of Genetic Medicine (G.V.R.), Johns Hopkins, Baltimore, MD; Division of Pediatric Blood and Marrow Transplantation & Cellular Therapy (T.L., P.J.O.), University of Minnesota, Minneapolis; Pediatric Oncology (J.-S.K.), Hematology, Hemostaseology, University Hospital Leipzig, Germany; Pediatric Blood and Bone Marrow Transplantation (C.A.L.), Princess Maxima Center Utrecht, the Netherlands; Department of Pediatrics (C.A.L.), Wilhemina Children's Hospital, UMC Utrecht, Utrecht University, the Netherlands; Director of Pediatric Neuroimaging (P.C.), Lenox Hill Radiology and Medical Imaging Associates, New York, NY; Moser Center for Leukodystrophies (B.R.T., A.F.), Kennedy Krieger Institute, Johns Hopkins Medical Institutions, Baltimore, MD; Department of Neurogenetics (A.B.M.), Kennedy Krieger Institute, Baltimore, MD; Laboratory Genetic Metabolic Diseases (F.M.V., S.F., S.K.), Department of Clinical Chemistry and Pediatrics, Amsterdam UMC, Amsterdam Gastroenterology Endocrinology Metabolism, University of Amsterdam, the Netherlands; and Department of Neurology (F.S.E.), Massachusetts General Hospital, Boston, MA. Dr. van Ballegoij is currently at the Department of Neurology, Zaans Medisch Centrum, Zaandam., van Trotsenburg ASP; From the Department of Pediatric Neurology/Emma Children's Hospital (M.E., W.J.C.B., I.C.H.), Amsterdam UMC, Amsterdam Leukodystrophy Center, University of Amsterdam, the Netherlands; Division of Child Neurology (E.J.M.), Department of Pediatrics, Weill Cornell Medicine/NewYork-Presbyterian Hospital, NY; Department of Neurology & Pediatrics/Lucile Packard Children's Hospital (K.P.V.H.), Stanford University School of Medicine, Palo Alto, CA 4. Department of Neurology, Leukodystrophy Clinic, University of Leipzig Medical Center, Germany; Unit of Rare Neurodegenerative and Neurometabolic Diseases (E.S.), Fondazione IRCCS Istituto Neurologico C. Besta, Milano, Italy; Department of Pediatric Endocrinology/Emma Children's Hospital (A.S.P.T.), Amsterdam UMC, University of Amsterdam, the Netherlands; AP-HP (F.M.), Department of Medical Genetics, Reference Center for Adult Neurometabolic Diseases and Leukodystrophies, and INSERM U 1127, CNRS UMR 7225, Paris Brain Institute, La Pitié-Salpêtrière University Hospital, Paris, France; Department of Pediatric Neurology/Hôpital Bicêtre Paris Sud (C.S.), France, Reference Center for Children Leukodystrophies Inserm U1127, ICM-Hôpital Pitié Salpêtrière, Paris, France; Division of Pediatric Endocrinology and Diabetes (M.O.R.), Children's Hospital at Montefiore, Albert Einstein College of Medicine of Medicine, Bronx, NY; Neuroendocrine Unit (N.A.T.), Massachusetts General Hospital, Boston, MA; Harvard Medical School (N.A.T.), Boston, MA; Division of Pediatric Endocrinology (A.H.), Department of Pediatrics, Massachusetts General Hospital, Boston, MA, and Harvard Medical School (A.H.), Boston, MA; Charles Dent Metabolic Unit (R.H.L.), National Hospital for Neurology and Neurosurgery, London, United Kingdom; Metabolic Medicine (J.D.), Great Ormond Street Hospital for Children, London United Kingdom; Department of Genetic Medicine (G.V.R.), Johns Hopkins, Baltimore, MD; Division of Pediatric Blood and Marrow Transplantation & Cellular Therapy (T.L., P.J.O.), University of Minnesota, Minneapolis; Pediatric Oncology (J.-S.K.), Hematology, Hemostaseology, University Hospital Leipzig, Germany; Pediatric Blood and Bone Marrow Transplantation (C.A.L.), Princess Maxima Center Utrecht, the Netherlands; Department of Pediatrics (C.A.L.), Wilhemina Children's Hospital, UMC Utrecht, Utrecht University, the Netherlands; Director of Pediatric Neuroimaging (P.C.), Lenox Hill Radiology and Medical Imaging Associates, New York, NY; Moser Center for Leukodystrophies (B.R.T., A.F.), Kennedy Krieger Institute, Johns Hopkins Medical Institutions, Baltimore, MD; Department of Neurogenetics (A.B.M.), Kennedy Krieger Institute, Baltimore, MD; Laboratory Genetic Metabolic Diseases (F.M.V., S.F., S.K.), Department of Clinical Chemistry and Pediatrics, Amsterdam UMC, Amsterdam Gastroenterology Endocrinology Metabolism, University of Amsterdam, the Netherlands; and Department of Neurology (F.S.E.), Massachusetts General Hospital, Boston, MA. Dr. van Ballegoij is currently at the Department of Neurology, Zaans Medisch Centrum, Zaandam., Mochel F; From the Department of Pediatric Neurology/Emma Children's Hospital (M.E., W.J.C.B., I.C.H.), Amsterdam UMC, Amsterdam Leukodystrophy Center, University of Amsterdam, the Netherlands; Division of Child Neurology (E.J.M.), Department of Pediatrics, Weill Cornell Medicine/NewYork-Presbyterian Hospital, NY; Department of Neurology & Pediatrics/Lucile Packard Children's Hospital (K.P.V.H.), Stanford University School of Medicine, Palo Alto, CA 4. Department of Neurology, Leukodystrophy Clinic, University of Leipzig Medical Center, Germany; Unit of Rare Neurodegenerative and Neurometabolic Diseases (E.S.), Fondazione IRCCS Istituto Neurologico C. Besta, Milano, Italy; Department of Pediatric Endocrinology/Emma Children's Hospital (A.S.P.T.), Amsterdam UMC, University of Amsterdam, the Netherlands; AP-HP (F.M.), Department of Medical Genetics, Reference Center for Adult Neurometabolic Diseases and Leukodystrophies, and INSERM U 1127, CNRS UMR 7225, Paris Brain Institute, La Pitié-Salpêtrière University Hospital, Paris, France; Department of Pediatric Neurology/Hôpital Bicêtre Paris Sud (C.S.), France, Reference Center for Children Leukodystrophies Inserm U1127, ICM-Hôpital Pitié Salpêtrière, Paris, France; Division of Pediatric Endocrinology and Diabetes (M.O.R.), Children's Hospital at Montefiore, Albert Einstein College of Medicine of Medicine, Bronx, NY; Neuroendocrine Unit (N.A.T.), Massachusetts General Hospital, Boston, MA; Harvard Medical School (N.A.T.), Boston, MA; Division of Pediatric Endocrinology (A.H.), Department of Pediatrics, Massachusetts General Hospital, Boston, MA, and Harvard Medical School (A.H.), Boston, MA; Charles Dent Metabolic Unit (R.H.L.), National Hospital for Neurology and Neurosurgery, London, United Kingdom; Metabolic Medicine (J.D.), Great Ormond Street Hospital for Children, London United Kingdom; Department of Genetic Medicine (G.V.R.), Johns Hopkins, Baltimore, MD; Division of Pediatric Blood and Marrow Transplantation & Cellular Therapy (T.L., P.J.O.), University of Minnesota, Minneapolis; Pediatric Oncology (J.-S.K.), Hematology, Hemostaseology, University Hospital Leipzig, Germany; Pediatric Blood and Bone Marrow Transplantation (C.A.L.), Princess Maxima Center Utrecht, the Netherlands; Department of Pediatrics (C.A.L.), Wilhemina Children's Hospital, UMC Utrecht, Utrecht University, the Netherlands; Director of Pediatric Neuroimaging (P.C.), Lenox Hill Radiology and Medical Imaging Associates, New York, NY; Moser Center for Leukodystrophies (B.R.T., A.F.), Kennedy Krieger Institute, Johns Hopkins Medical Institutions, Baltimore, MD; Department of Neurogenetics (A.B.M.), Kennedy Krieger Institute, Baltimore, MD; Laboratory Genetic Metabolic Diseases (F.M.V., S.F., S.K.), Department of Clinical Chemistry and Pediatrics, Amsterdam UMC, Amsterdam Gastroenterology Endocrinology Metabolism, University of Amsterdam, the Netherlands; and Department of Neurology (F.S.E.), Massachusetts General Hospital, Boston, MA. Dr. van Ballegoij is currently at the Department of Neurology, Zaans Medisch Centrum, Zaandam., Sevin C; From the Department of Pediatric Neurology/Emma Children's Hospital (M.E., W.J.C.B., I.C.H.), Amsterdam UMC, Amsterdam Leukodystrophy Center, University of Amsterdam, the Netherlands; Division of Child Neurology (E.J.M.), Department of Pediatrics, Weill Cornell Medicine/NewYork-Presbyterian Hospital, NY; Department of Neurology & Pediatrics/Lucile Packard Children's Hospital (K.P.V.H.), Stanford University School of Medicine, Palo Alto, CA 4. Department of Neurology, Leukodystrophy Clinic, University of Leipzig Medical Center, Germany; Unit of Rare Neurodegenerative and Neurometabolic Diseases (E.S.), Fondazione IRCCS Istituto Neurologico C. Besta, Milano, Italy; Department of Pediatric Endocrinology/Emma Children's Hospital (A.S.P.T.), Amsterdam UMC, University of Amsterdam, the Netherlands; AP-HP (F.M.), Department of Medical Genetics, Reference Center for Adult Neurometabolic Diseases and Leukodystrophies, and INSERM U 1127, CNRS UMR 7225, Paris Brain Institute, La Pitié-Salpêtrière University Hospital, Paris, France; Department of Pediatric Neurology/Hôpital Bicêtre Paris Sud (C.S.), France, Reference Center for Children Leukodystrophies Inserm U1127, ICM-Hôpital Pitié Salpêtrière, Paris, France; Division of Pediatric Endocrinology and Diabetes (M.O.R.), Children's Hospital at Montefiore, Albert Einstein College of Medicine of Medicine, Bronx, NY; Neuroendocrine Unit (N.A.T.), Massachusetts General Hospital, Boston, MA; Harvard Medical School (N.A.T.), Boston, MA; Division of Pediatric Endocrinology (A.H.), Department of Pediatrics, Massachusetts General Hospital, Boston, MA, and Harvard Medical School (A.H.), Boston, MA; Charles Dent Metabolic Unit (R.H.L.), National Hospital for Neurology and Neurosurgery, London, United Kingdom; Metabolic Medicine (J.D.), Great Ormond Street Hospital for Children, London United Kingdom; Department of Genetic Medicine (G.V.R.), Johns Hopkins, Baltimore, MD; Division of Pediatric Blood and Marrow Transplantation & Cellular Therapy (T.L., P.J.O.), University of Minnesota, Minneapolis; Pediatric Oncology (J.-S.K.), Hematology, Hemostaseology, University Hospital Leipzig, Germany; Pediatric Blood and Bone Marrow Transplantation (C.A.L.), Princess Maxima Center Utrecht, the Netherlands; Department of Pediatrics (C.A.L.), Wilhemina Children's Hospital, UMC Utrecht, Utrecht University, the Netherlands; Director of Pediatric Neuroimaging (P.C.), Lenox Hill Radiology and Medical Imaging Associates, New York, NY; Moser Center for Leukodystrophies (B.R.T., A.F.), Kennedy Krieger Institute, Johns Hopkins Medical Institutions, Baltimore, MD; Department of Neurogenetics (A.B.M.), Kennedy Krieger Institute, Baltimore, MD; Laboratory Genetic Metabolic Diseases (F.M.V., S.F., S.K.), Department of Clinical Chemistry and Pediatrics, Amsterdam UMC, Amsterdam Gastroenterology Endocrinology Metabolism, University of Amsterdam, the Netherlands; and Department of Neurology (F.S.E.), Massachusetts General Hospital, Boston, MA. Dr. van Ballegoij is currently at the Department of Neurology, Zaans Medisch Centrum, Zaandam., Regelmann MO; From the Department of Pediatric Neurology/Emma Children's Hospital (M.E., W.J.C.B., I.C.H.), Amsterdam UMC, Amsterdam Leukodystrophy Center, University of Amsterdam, the Netherlands; Division of Child Neurology (E.J.M.), Department of Pediatrics, Weill Cornell Medicine/NewYork-Presbyterian Hospital, NY; Department of Neurology & Pediatrics/Lucile Packard Children's Hospital (K.P.V.H.), Stanford University School of Medicine, Palo Alto, CA 4. Department of Neurology, Leukodystrophy Clinic, University of Leipzig Medical Center, Germany; Unit of Rare Neurodegenerative and Neurometabolic Diseases (E.S.), Fondazione IRCCS Istituto Neurologico C. Besta, Milano, Italy; Department of Pediatric Endocrinology/Emma Children's Hospital (A.S.P.T.), Amsterdam UMC, University of Amsterdam, the Netherlands; AP-HP (F.M.), Department of Medical Genetics, Reference Center for Adult Neurometabolic Diseases and Leukodystrophies, and INSERM U 1127, CNRS UMR 7225, Paris Brain Institute, La Pitié-Salpêtrière University Hospital, Paris, France; Department of Pediatric Neurology/Hôpital Bicêtre Paris Sud (C.S.), France, Reference Center for Children Leukodystrophies Inserm U1127, ICM-Hôpital Pitié Salpêtrière, Paris, France; Division of Pediatric Endocrinology and Diabetes (M.O.R.), Children's Hospital at Montefiore, Albert Einstein College of Medicine of Medicine, Bronx, NY; Neuroendocrine Unit (N.A.T.), Massachusetts General Hospital, Boston, MA; Harvard Medical School (N.A.T.), Boston, MA; Division of Pediatric Endocrinology (A.H.), Department of Pediatrics, Massachusetts General Hospital, Boston, MA, and Harvard Medical School (A.H.), Boston, MA; Charles Dent Metabolic Unit (R.H.L.), National Hospital for Neurology and Neurosurgery, London, United Kingdom; Metabolic Medicine (J.D.), Great Ormond Street Hospital for Children, London United Kingdom; Department of Genetic Medicine (G.V.R.), Johns Hopkins, Baltimore, MD; Division of Pediatric Blood and Marrow Transplantation & Cellular Therapy (T.L., P.J.O.), University of Minnesota, Minneapolis; Pediatric Oncology (J.-S.K.), Hematology, Hemostaseology, University Hospital Leipzig, Germany; Pediatric Blood and Bone Marrow Transplantation (C.A.L.), Princess Maxima Center Utrecht, the Netherlands; Department of Pediatrics (C.A.L.), Wilhemina Children's Hospital, UMC Utrecht, Utrecht University, the Netherlands; Director of Pediatric Neuroimaging (P.C.), Lenox Hill Radiology and Medical Imaging Associates, New York, NY; Moser Center for Leukodystrophies (B.R.T., A.F.), Kennedy Krieger Institute, Johns Hopkins Medical Institutions, Baltimore, MD; Department of Neurogenetics (A.B.M.), Kennedy Krieger Institute, Baltimore, MD; Laboratory Genetic Metabolic Diseases (F.M.V., S.F., S.K.), Department of Clinical Chemistry and Pediatrics, Amsterdam UMC, Amsterdam Gastroenterology Endocrinology Metabolism, University of Amsterdam, the Netherlands; and Department of Neurology (F.S.E.), Massachusetts General Hospital, Boston, MA. Dr. van Ballegoij is currently at the Department of Neurology, Zaans Medisch Centrum, Zaandam., Tritos NA; From the Department of Pediatric Neurology/Emma Children's Hospital (M.E., W.J.C.B., I.C.H.), Amsterdam UMC, Amsterdam Leukodystrophy Center, University of Amsterdam, the Netherlands; Division of Child Neurology (E.J.M.), Department of Pediatrics, Weill Cornell Medicine/NewYork-Presbyterian Hospital, NY; Department of Neurology & Pediatrics/Lucile Packard Children's Hospital (K.P.V.H.), Stanford University School of Medicine, Palo Alto, CA 4. Department of Neurology, Leukodystrophy Clinic, University of Leipzig Medical Center, Germany; Unit of Rare Neurodegenerative and Neurometabolic Diseases (E.S.), Fondazione IRCCS Istituto Neurologico C. Besta, Milano, Italy; Department of Pediatric Endocrinology/Emma Children's Hospital (A.S.P.T.), Amsterdam UMC, University of Amsterdam, the Netherlands; AP-HP (F.M.), Department of Medical Genetics, Reference Center for Adult Neurometabolic Diseases and Leukodystrophies, and INSERM U 1127, CNRS UMR 7225, Paris Brain Institute, La Pitié-Salpêtrière University Hospital, Paris, France; Department of Pediatric Neurology/Hôpital Bicêtre Paris Sud (C.S.), France, Reference Center for Children Leukodystrophies Inserm U1127, ICM-Hôpital Pitié Salpêtrière, Paris, France; Division of Pediatric Endocrinology and Diabetes (M.O.R.), Children's Hospital at Montefiore, Albert Einstein College of Medicine of Medicine, Bronx, NY; Neuroendocrine Unit (N.A.T.), Massachusetts General Hospital, Boston, MA; Harvard Medical School (N.A.T.), Boston, MA; Division of Pediatric Endocrinology (A.H.), Department of Pediatrics, Massachusetts General Hospital, Boston, MA, and Harvard Medical School (A.H.), Boston, MA; Charles Dent Metabolic Unit (R.H.L.), National Hospital for Neurology and Neurosurgery, London, United Kingdom; Metabolic Medicine (J.D.), Great Ormond Street Hospital for Children, London United Kingdom; Department of Genetic Medicine (G.V.R.), Johns Hopkins, Baltimore, MD; Division of Pediatric Blood and Marrow Transplantation & Cellular Therapy (T.L., P.J.O.), University of Minnesota, Minneapolis; Pediatric Oncology (J.-S.K.), Hematology, Hemostaseology, University Hospital Leipzig, Germany; Pediatric Blood and Bone Marrow Transplantation (C.A.L.), Princess Maxima Center Utrecht, the Netherlands; Department of Pediatrics (C.A.L.), Wilhemina Children's Hospital, UMC Utrecht, Utrecht University, the Netherlands; Director of Pediatric Neuroimaging (P.C.), Lenox Hill Radiology and Medical Imaging Associates, New York, NY; Moser Center for Leukodystrophies (B.R.T., A.F.), Kennedy Krieger Institute, Johns Hopkins Medical Institutions, Baltimore, MD; Department of Neurogenetics (A.B.M.), Kennedy Krieger Institute, Baltimore, MD; Laboratory Genetic Metabolic Diseases (F.M.V., S.F., S.K.), Department of Clinical Chemistry and Pediatrics, Amsterdam UMC, Amsterdam Gastroenterology Endocrinology Metabolism, University of Amsterdam, the Netherlands; and Department of Neurology (F.S.E.), Massachusetts General Hospital, Boston, MA. Dr. van Ballegoij is currently at the Department of Neurology, Zaans Medisch Centrum, Zaandam., Halper A; From the Department of Pediatric Neurology/Emma Children's Hospital (M.E., W.J.C.B., I.C.H.), Amsterdam UMC, Amsterdam Leukodystrophy Center, University of Amsterdam, the Netherlands; Division of Child Neurology (E.J.M.), Department of Pediatrics, Weill Cornell Medicine/NewYork-Presbyterian Hospital, NY; Department of Neurology & Pediatrics/Lucile Packard Children's Hospital (K.P.V.H.), Stanford University School of Medicine, Palo Alto, CA 4. Department of Neurology, Leukodystrophy Clinic, University of Leipzig Medical Center, Germany; Unit of Rare Neurodegenerative and Neurometabolic Diseases (E.S.), Fondazione IRCCS Istituto Neurologico C. Besta, Milano, Italy; Department of Pediatric Endocrinology/Emma Children's Hospital (A.S.P.T.), Amsterdam UMC, University of Amsterdam, the Netherlands; AP-HP (F.M.), Department of Medical Genetics, Reference Center for Adult Neurometabolic Diseases and Leukodystrophies, and INSERM U 1127, CNRS UMR 7225, Paris Brain Institute, La Pitié-Salpêtrière University Hospital, Paris, France; Department of Pediatric Neurology/Hôpital Bicêtre Paris Sud (C.S.), France, Reference Center for Children Leukodystrophies Inserm U1127, ICM-Hôpital Pitié Salpêtrière, Paris, France; Division of Pediatric Endocrinology and Diabetes (M.O.R.), Children's Hospital at Montefiore, Albert Einstein College of Medicine of Medicine, Bronx, NY; Neuroendocrine Unit (N.A.T.), Massachusetts General Hospital, Boston, MA; Harvard Medical School (N.A.T.), Boston, MA; Division of Pediatric Endocrinology (A.H.), Department of Pediatrics, Massachusetts General Hospital, Boston, MA, and Harvard Medical School (A.H.), Boston, MA; Charles Dent Metabolic Unit (R.H.L.), National Hospital for Neurology and Neurosurgery, London, United Kingdom; Metabolic Medicine (J.D.), Great Ormond Street Hospital for Children, London United Kingdom; Department of Genetic Medicine (G.V.R.), Johns Hopkins, Baltimore, MD; Division of Pediatric Blood and Marrow Transplantation & Cellular Therapy (T.L., P.J.O.), University of Minnesota, Minneapolis; Pediatric Oncology (J.-S.K.), Hematology, Hemostaseology, University Hospital Leipzig, Germany; Pediatric Blood and Bone Marrow Transplantation (C.A.L.), Princess Maxima Center Utrecht, the Netherlands; Department of Pediatrics (C.A.L.), Wilhemina Children's Hospital, UMC Utrecht, Utrecht University, the Netherlands; Director of Pediatric Neuroimaging (P.C.), Lenox Hill Radiology and Medical Imaging Associates, New York, NY; Moser Center for Leukodystrophies (B.R.T., A.F.), Kennedy Krieger Institute, Johns Hopkins Medical Institutions, Baltimore, MD; Department of Neurogenetics (A.B.M.), Kennedy Krieger Institute, Baltimore, MD; Laboratory Genetic Metabolic Diseases (F.M.V., S.F., S.K.), Department of Clinical Chemistry and Pediatrics, Amsterdam UMC, Amsterdam Gastroenterology Endocrinology Metabolism, University of Amsterdam, the Netherlands; and Department of Neurology (F.S.E.), Massachusetts General Hospital, Boston, MA. Dr. van Ballegoij is currently at the Department of Neurology, Zaans Medisch Centrum, Zaandam., Lachmann RH; From the Department of Pediatric Neurology/Emma Children's Hospital (M.E., W.J.C.B., I.C.H.), Amsterdam UMC, Amsterdam Leukodystrophy Center, University of Amsterdam, the Netherlands; Division of Child Neurology (E.J.M.), Department of Pediatrics, Weill Cornell Medicine/NewYork-Presbyterian Hospital, NY; Department of Neurology & Pediatrics/Lucile Packard Children's Hospital (K.P.V.H.), Stanford University School of Medicine, Palo Alto, CA 4. Department of Neurology, Leukodystrophy Clinic, University of Leipzig Medical Center, Germany; Unit of Rare Neurodegenerative and Neurometabolic Diseases (E.S.), Fondazione IRCCS Istituto Neurologico C. Besta, Milano, Italy; Department of Pediatric Endocrinology/Emma Children's Hospital (A.S.P.T.), Amsterdam UMC, University of Amsterdam, the Netherlands; AP-HP (F.M.), Department of Medical Genetics, Reference Center for Adult Neurometabolic Diseases and Leukodystrophies, and INSERM U 1127, CNRS UMR 7225, Paris Brain Institute, La Pitié-Salpêtrière University Hospital, Paris, France; Department of Pediatric Neurology/Hôpital Bicêtre Paris Sud (C.S.), France, Reference Center for Children Leukodystrophies Inserm U1127, ICM-Hôpital Pitié Salpêtrière, Paris, France; Division of Pediatric Endocrinology and Diabetes (M.O.R.), Children's Hospital at Montefiore, Albert Einstein College of Medicine of Medicine, Bronx, NY; Neuroendocrine Unit (N.A.T.), Massachusetts General Hospital, Boston, MA; Harvard Medical School (N.A.T.), Boston, MA; Division of Pediatric Endocrinology (A.H.), Department of Pediatrics, Massachusetts General Hospital, Boston, MA, and Harvard Medical School (A.H.), Boston, MA; Charles Dent Metabolic Unit (R.H.L.), National Hospital for Neurology and Neurosurgery, London, United Kingdom; Metabolic Medicine (J.D.), Great Ormond Street Hospital for Children, London United Kingdom; Department of Genetic Medicine (G.V.R.), Johns Hopkins, Baltimore, MD; Division of Pediatric Blood and Marrow Transplantation & Cellular Therapy (T.L., P.J.O.), University of Minnesota, Minneapolis; Pediatric Oncology (J.-S.K.), Hematology, Hemostaseology, University Hospital Leipzig, Germany; Pediatric Blood and Bone Marrow Transplantation (C.A.L.), Princess Maxima Center Utrecht, the Netherlands; Department of Pediatrics (C.A.L.), Wilhemina Children's Hospital, UMC Utrecht, Utrecht University, the Netherlands; Director of Pediatric Neuroimaging (P.C.), Lenox Hill Radiology and Medical Imaging Associates, New York, NY; Moser Center for Leukodystrophies (B.R.T., A.F.), Kennedy Krieger Institute, Johns Hopkins Medical Institutions, Baltimore, MD; Department of Neurogenetics (A.B.M.), Kennedy Krieger Institute, Baltimore, MD; Laboratory Genetic Metabolic Diseases (F.M.V., S.F., S.K.), Department of Clinical Chemistry and Pediatrics, Amsterdam UMC, Amsterdam Gastroenterology Endocrinology Metabolism, University of Amsterdam, the Netherlands; and Department of Neurology (F.S.E.), Massachusetts General Hospital, Boston, MA. Dr. van Ballegoij is currently at the Department of Neurology, Zaans Medisch Centrum, Zaandam., Davison J; From the Department of Pediatric Neurology/Emma Children's Hospital (M.E., W.J.C.B., I.C.H.), Amsterdam UMC, Amsterdam Leukodystrophy Center, University of Amsterdam, the Netherlands; Division of Child Neurology (E.J.M.), Department of Pediatrics, Weill Cornell Medicine/NewYork-Presbyterian Hospital, NY; Department of Neurology & Pediatrics/Lucile Packard Children's Hospital (K.P.V.H.), Stanford University School of Medicine, Palo Alto, CA 4. Department of Neurology, Leukodystrophy Clinic, University of Leipzig Medical Center, Germany; Unit of Rare Neurodegenerative and Neurometabolic Diseases (E.S.), Fondazione IRCCS Istituto Neurologico C. Besta, Milano, Italy; Department of Pediatric Endocrinology/Emma Children's Hospital (A.S.P.T.), Amsterdam UMC, University of Amsterdam, the Netherlands; AP-HP (F.M.), Department of Medical Genetics, Reference Center for Adult Neurometabolic Diseases and Leukodystrophies, and INSERM U 1127, CNRS UMR 7225, Paris Brain Institute, La Pitié-Salpêtrière University Hospital, Paris, France; Department of Pediatric Neurology/Hôpital Bicêtre Paris Sud (C.S.), France, Reference Center for Children Leukodystrophies Inserm U1127, ICM-Hôpital Pitié Salpêtrière, Paris, France; Division of Pediatric Endocrinology and Diabetes (M.O.R.), Children's Hospital at Montefiore, Albert Einstein College of Medicine of Medicine, Bronx, NY; Neuroendocrine Unit (N.A.T.), Massachusetts General Hospital, Boston, MA; Harvard Medical School (N.A.T.), Boston, MA; Division of Pediatric Endocrinology (A.H.), Department of Pediatrics, Massachusetts General Hospital, Boston, MA, and Harvard Medical School (A.H.), Boston, MA; Charles Dent Metabolic Unit (R.H.L.), National Hospital for Neurology and Neurosurgery, London, United Kingdom; Metabolic Medicine (J.D.), Great Ormond Street Hospital for Children, London United Kingdom; Department of Genetic Medicine (G.V.R.), Johns Hopkins, Baltimore, MD; Division of Pediatric Blood and Marrow Transplantation & Cellular Therapy (T.L., P.J.O.), University of Minnesota, Minneapolis; Pediatric Oncology (J.-S.K.), Hematology, Hemostaseology, University Hospital Leipzig, Germany; Pediatric Blood and Bone Marrow Transplantation (C.A.L.), Princess Maxima Center Utrecht, the Netherlands; Department of Pediatrics (C.A.L.), Wilhemina Children's Hospital, UMC Utrecht, Utrecht University, the Netherlands; Director of Pediatric Neuroimaging (P.C.), Lenox Hill Radiology and Medical Imaging Associates, New York, NY; Moser Center for Leukodystrophies (B.R.T., A.F.), Kennedy Krieger Institute, Johns Hopkins Medical Institutions, Baltimore, MD; Department of Neurogenetics (A.B.M.), Kennedy Krieger Institute, Baltimore, MD; Laboratory Genetic Metabolic Diseases (F.M.V., S.F., S.K.), Department of Clinical Chemistry and Pediatrics, Amsterdam UMC, Amsterdam Gastroenterology Endocrinology Metabolism, University of Amsterdam, the Netherlands; and Department of Neurology (F.S.E.), Massachusetts General Hospital, Boston, MA. Dr. van Ballegoij is currently at the Department of Neurology, Zaans Medisch Centrum, Zaandam., Raymond GV; From the Department of Pediatric Neurology/Emma Children's Hospital (M.E., W.J.C.B., I.C.H.), Amsterdam UMC, Amsterdam Leukodystrophy Center, University of Amsterdam, the Netherlands; Division of Child Neurology (E.J.M.), Department of Pediatrics, Weill Cornell Medicine/NewYork-Presbyterian Hospital, NY; Department of Neurology & Pediatrics/Lucile Packard Children's Hospital (K.P.V.H.), Stanford University School of Medicine, Palo Alto, CA 4. Department of Neurology, Leukodystrophy Clinic, University of Leipzig Medical Center, Germany; Unit of Rare Neurodegenerative and Neurometabolic Diseases (E.S.), Fondazione IRCCS Istituto Neurologico C. Besta, Milano, Italy; Department of Pediatric Endocrinology/Emma Children's Hospital (A.S.P.T.), Amsterdam UMC, University of Amsterdam, the Netherlands; AP-HP (F.M.), Department of Medical Genetics, Reference Center for Adult Neurometabolic Diseases and Leukodystrophies, and INSERM U 1127, CNRS UMR 7225, Paris Brain Institute, La Pitié-Salpêtrière University Hospital, Paris, France; Department of Pediatric Neurology/Hôpital Bicêtre Paris Sud (C.S.), France, Reference Center for Children Leukodystrophies Inserm U1127, ICM-Hôpital Pitié Salpêtrière, Paris, France; Division of Pediatric Endocrinology and Diabetes (M.O.R.), Children's Hospital at Montefiore, Albert Einstein College of Medicine of Medicine, Bronx, NY; Neuroendocrine Unit (N.A.T.), Massachusetts General Hospital, Boston, MA; Harvard Medical School (N.A.T.), Boston, MA; Division of Pediatric Endocrinology (A.H.), Department of Pediatrics, Massachusetts General Hospital, Boston, MA, and Harvard Medical School (A.H.), Boston, MA; Charles Dent Metabolic Unit (R.H.L.), National Hospital for Neurology and Neurosurgery, London, United Kingdom; Metabolic Medicine (J.D.), Great Ormond Street Hospital for Children, London United Kingdom; Department of Genetic Medicine (G.V.R.), Johns Hopkins, Baltimore, MD; Division of Pediatric Blood and Marrow Transplantation & Cellular Therapy (T.L., P.J.O.), University of Minnesota, Minneapolis; Pediatric Oncology (J.-S.K.), Hematology, Hemostaseology, University Hospital Leipzig, Germany; Pediatric Blood and Bone Marrow Transplantation (C.A.L.), Princess Maxima Center Utrecht, the Netherlands; Department of Pediatrics (C.A.L.), Wilhemina Children's Hospital, UMC Utrecht, Utrecht University, the Netherlands; Director of Pediatric Neuroimaging (P.C.), Lenox Hill Radiology and Medical Imaging Associates, New York, NY; Moser Center for Leukodystrophies (B.R.T., A.F.), Kennedy Krieger Institute, Johns Hopkins Medical Institutions, Baltimore, MD; Department of Neurogenetics (A.B.M.), Kennedy Krieger Institute, Baltimore, MD; Laboratory Genetic Metabolic Diseases (F.M.V., S.F., S.K.), Department of Clinical Chemistry and Pediatrics, Amsterdam UMC, Amsterdam Gastroenterology Endocrinology Metabolism, University of Amsterdam, the Netherlands; and Department of Neurology (F.S.E.), Massachusetts General Hospital, Boston, MA. Dr. van Ballegoij is currently at the Department of Neurology, Zaans Medisch Centrum, Zaandam., Lund TC; From the Department of Pediatric Neurology/Emma Children's Hospital (M.E., W.J.C.B., I.C.H.), Amsterdam UMC, Amsterdam Leukodystrophy Center, University of Amsterdam, the Netherlands; Division of Child Neurology (E.J.M.), Department of Pediatrics, Weill Cornell Medicine/NewYork-Presbyterian Hospital, NY; Department of Neurology & Pediatrics/Lucile Packard Children's Hospital (K.P.V.H.), Stanford University School of Medicine, Palo Alto, CA 4. Department of Neurology, Leukodystrophy Clinic, University of Leipzig Medical Center, Germany; Unit of Rare Neurodegenerative and Neurometabolic Diseases (E.S.), Fondazione IRCCS Istituto Neurologico C. Besta, Milano, Italy; Department of Pediatric Endocrinology/Emma Children's Hospital (A.S.P.T.), Amsterdam UMC, University of Amsterdam, the Netherlands; AP-HP (F.M.), Department of Medical Genetics, Reference Center for Adult Neurometabolic Diseases and Leukodystrophies, and INSERM U 1127, CNRS UMR 7225, Paris Brain Institute, La Pitié-Salpêtrière University Hospital, Paris, France; Department of Pediatric Neurology/Hôpital Bicêtre Paris Sud (C.S.), France, Reference Center for Children Leukodystrophies Inserm U1127, ICM-Hôpital Pitié Salpêtrière, Paris, France; Division of Pediatric Endocrinology and Diabetes (M.O.R.), Children's Hospital at Montefiore, Albert Einstein College of Medicine of Medicine, Bronx, NY; Neuroendocrine Unit (N.A.T.), Massachusetts General Hospital, Boston, MA; Harvard Medical School (N.A.T.), Boston, MA; Division of Pediatric Endocrinology (A.H.), Department of Pediatrics, Massachusetts General Hospital, Boston, MA, and Harvard Medical School (A.H.), Boston, MA; Charles Dent Metabolic Unit (R.H.L.), National Hospital for Neurology and Neurosurgery, London, United Kingdom; Metabolic Medicine (J.D.), Great Ormond Street Hospital for Children, London United Kingdom; Department of Genetic Medicine (G.V.R.), Johns Hopkins, Baltimore, MD; Division of Pediatric Blood and Marrow Transplantation & Cellular Therapy (T.L., P.J.O.), University of Minnesota, Minneapolis; Pediatric Oncology (J.-S.K.), Hematology, Hemostaseology, University Hospital Leipzig, Germany; Pediatric Blood and Bone Marrow Transplantation (C.A.L.), Princess Maxima Center Utrecht, the Netherlands; Department of Pediatrics (C.A.L.), Wilhemina Children's Hospital, UMC Utrecht, Utrecht University, the Netherlands; Director of Pediatric Neuroimaging (P.C.), Lenox Hill Radiology and Medical Imaging Associates, New York, NY; Moser Center for Leukodystrophies (B.R.T., A.F.), Kennedy Krieger Institute, Johns Hopkins Medical Institutions, Baltimore, MD; Department of Neurogenetics (A.B.M.), Kennedy Krieger Institute, Baltimore, MD; Laboratory Genetic Metabolic Diseases (F.M.V., S.F., S.K.), Department of Clinical Chemistry and Pediatrics, Amsterdam UMC, Amsterdam Gastroenterology Endocrinology Metabolism, University of Amsterdam, the Netherlands; and Department of Neurology (F.S.E.), Massachusetts General Hospital, Boston, MA. Dr. van Ballegoij is currently at the Department of Neurology, Zaans Medisch Centrum, Zaandam., Orchard PJ; From the Department of Pediatric Neurology/Emma Children's Hospital (M.E., W.J.C.B., I.C.H.), Amsterdam UMC, Amsterdam Leukodystrophy Center, University of Amsterdam, the Netherlands; Division of Child Neurology (E.J.M.), Department of Pediatrics, Weill Cornell Medicine/NewYork-Presbyterian Hospital, NY; Department of Neurology & Pediatrics/Lucile Packard Children's Hospital (K.P.V.H.), Stanford University School of Medicine, Palo Alto, CA 4. Department of Neurology, Leukodystrophy Clinic, University of Leipzig Medical Center, Germany; Unit of Rare Neurodegenerative and Neurometabolic Diseases (E.S.), Fondazione IRCCS Istituto Neurologico C. Besta, Milano, Italy; Department of Pediatric Endocrinology/Emma Children's Hospital (A.S.P.T.), Amsterdam UMC, University of Amsterdam, the Netherlands; AP-HP (F.M.), Department of Medical Genetics, Reference Center for Adult Neurometabolic Diseases and Leukodystrophies, and INSERM U 1127, CNRS UMR 7225, Paris Brain Institute, La Pitié-Salpêtrière University Hospital, Paris, France; Department of Pediatric Neurology/Hôpital Bicêtre Paris Sud (C.S.), France, Reference Center for Children Leukodystrophies Inserm U1127, ICM-Hôpital Pitié Salpêtrière, Paris, France; Division of Pediatric Endocrinology and Diabetes (M.O.R.), Children's Hospital at Montefiore, Albert Einstein College of Medicine of Medicine, Bronx, NY; Neuroendocrine Unit (N.A.T.), Massachusetts General Hospital, Boston, MA; Harvard Medical School (N.A.T.), Boston, MA; Division of Pediatric Endocrinology (A.H.), Department of Pediatrics, Massachusetts General Hospital, Boston, MA, and Harvard Medical School (A.H.), Boston, MA; Charles Dent Metabolic Unit (R.H.L.), National Hospital for Neurology and Neurosurgery, London, United Kingdom; Metabolic Medicine (J.D.), Great Ormond Street Hospital for Children, London United Kingdom; Department of Genetic Medicine (G.V.R.), Johns Hopkins, Baltimore, MD; Division of Pediatric Blood and Marrow Transplantation & Cellular Therapy (T.L., P.J.O.), University of Minnesota, Minneapolis; Pediatric Oncology (J.-S.K.), Hematology, Hemostaseology, University Hospital Leipzig, Germany; Pediatric Blood and Bone Marrow Transplantation (C.A.L.), Princess Maxima Center Utrecht, the Netherlands; Department of Pediatrics (C.A.L.), Wilhemina Children's Hospital, UMC Utrecht, Utrecht University, the Netherlands; Director of Pediatric Neuroimaging (P.C.), Lenox Hill Radiology and Medical Imaging Associates, New York, NY; Moser Center for Leukodystrophies (B.R.T., A.F.), Kennedy Krieger Institute, Johns Hopkins Medical Institutions, Baltimore, MD; Department of Neurogenetics (A.B.M.), Kennedy Krieger Institute, Baltimore, MD; Laboratory Genetic Metabolic Diseases (F.M.V., S.F., S.K.), Department of Clinical Chemistry and Pediatrics, Amsterdam UMC, Amsterdam Gastroenterology Endocrinology Metabolism, University of Amsterdam, the Netherlands; and Department of Neurology (F.S.E.), Massachusetts General Hospital, Boston, MA. Dr. van Ballegoij is currently at the Department of Neurology, Zaans Medisch Centrum, Zaandam., Kuehl JS; From the Department of Pediatric Neurology/Emma Children's Hospital (M.E., W.J.C.B., I.C.H.), Amsterdam UMC, Amsterdam Leukodystrophy Center, University of Amsterdam, the Netherlands; Division of Child Neurology (E.J.M.), Department of Pediatrics, Weill Cornell Medicine/NewYork-Presbyterian Hospital, NY; Department of Neurology & Pediatrics/Lucile Packard Children's Hospital (K.P.V.H.), Stanford University School of Medicine, Palo Alto, CA 4. Department of Neurology, Leukodystrophy Clinic, University of Leipzig Medical Center, Germany; Unit of Rare Neurodegenerative and Neurometabolic Diseases (E.S.), Fondazione IRCCS Istituto Neurologico C. Besta, Milano, Italy; Department of Pediatric Endocrinology/Emma Children's Hospital (A.S.P.T.), Amsterdam UMC, University of Amsterdam, the Netherlands; AP-HP (F.M.), Department of Medical Genetics, Reference Center for Adult Neurometabolic Diseases and Leukodystrophies, and INSERM U 1127, CNRS UMR 7225, Paris Brain Institute, La Pitié-Salpêtrière University Hospital, Paris, France; Department of Pediatric Neurology/Hôpital Bicêtre Paris Sud (C.S.), France, Reference Center for Children Leukodystrophies Inserm U1127, ICM-Hôpital Pitié Salpêtrière, Paris, France; Division of Pediatric Endocrinology and Diabetes (M.O.R.), Children's Hospital at Montefiore, Albert Einstein College of Medicine of Medicine, Bronx, NY; Neuroendocrine Unit (N.A.T.), Massachusetts General Hospital, Boston, MA; Harvard Medical School (N.A.T.), Boston, MA; Division of Pediatric Endocrinology (A.H.), Department of Pediatrics, Massachusetts General Hospital, Boston, MA, and Harvard Medical School (A.H.), Boston, MA; Charles Dent Metabolic Unit (R.H.L.), National Hospital for Neurology and Neurosurgery, London, United Kingdom; Metabolic Medicine (J.D.), Great Ormond Street Hospital for Children, London United Kingdom; Department of Genetic Medicine (G.V.R.), Johns Hopkins, Baltimore, MD; Division of Pediatric Blood and Marrow Transplantation & Cellular Therapy (T.L., P.J.O.), University of Minnesota, Minneapolis; Pediatric Oncology (J.-S.K.), Hematology, Hemostaseology, University Hospital Leipzig, Germany; Pediatric Blood and Bone Marrow Transplantation (C.A.L.), Princess Maxima Center Utrecht, the Netherlands; Department of Pediatrics (C.A.L.), Wilhemina Children's Hospital, UMC Utrecht, Utrecht University, the Netherlands; Director of Pediatric Neuroimaging (P.C.), Lenox Hill Radiology and Medical Imaging Associates, New York, NY; Moser Center for Leukodystrophies (B.R.T., A.F.), Kennedy Krieger Institute, Johns Hopkins Medical Institutions, Baltimore, MD; Department of Neurogenetics (A.B.M.), Kennedy Krieger Institute, Baltimore, MD; Laboratory Genetic Metabolic Diseases (F.M.V., S.F., S.K.), Department of Clinical Chemistry and Pediatrics, Amsterdam UMC, Amsterdam Gastroenterology Endocrinology Metabolism, University of Amsterdam, the Netherlands; and Department of Neurology (F.S.E.), Massachusetts General Hospital, Boston, MA. Dr. van Ballegoij is currently at the Department of Neurology, Zaans Medisch Centrum, Zaandam., Lindemans CA; From the Department of Pediatric Neurology/Emma Children's Hospital (M.E., W.J.C.B., I.C.H.), Amsterdam UMC, Amsterdam Leukodystrophy Center, University of Amsterdam, the Netherlands; Division of Child Neurology (E.J.M.), Department of Pediatrics, Weill Cornell Medicine/NewYork-Presbyterian Hospital, NY; Department of Neurology & Pediatrics/Lucile Packard Children's Hospital (K.P.V.H.), Stanford University School of Medicine, Palo Alto, CA 4. Department of Neurology, Leukodystrophy Clinic, University of Leipzig Medical Center, Germany; Unit of Rare Neurodegenerative and Neurometabolic Diseases (E.S.), Fondazione IRCCS Istituto Neurologico C. Besta, Milano, Italy; Department of Pediatric Endocrinology/Emma Children's Hospital (A.S.P.T.), Amsterdam UMC, University of Amsterdam, the Netherlands; AP-HP (F.M.), Department of Medical Genetics, Reference Center for Adult Neurometabolic Diseases and Leukodystrophies, and INSERM U 1127, CNRS UMR 7225, Paris Brain Institute, La Pitié-Salpêtrière University Hospital, Paris, France; Department of Pediatric Neurology/Hôpital Bicêtre Paris Sud (C.S.), France, Reference Center for Children Leukodystrophies Inserm U1127, ICM-Hôpital Pitié Salpêtrière, Paris, France; Division of Pediatric Endocrinology and Diabetes (M.O.R.), Children's Hospital at Montefiore, Albert Einstein College of Medicine of Medicine, Bronx, NY; Neuroendocrine Unit (N.A.T.), Massachusetts General Hospital, Boston, MA; Harvard Medical School (N.A.T.), Boston, MA; Division of Pediatric Endocrinology (A.H.), Department of Pediatrics, Massachusetts General Hospital, Boston, MA, and Harvard Medical School (A.H.), Boston, MA; Charles Dent Metabolic Unit (R.H.L.), National Hospital for Neurology and Neurosurgery, London, United Kingdom; Metabolic Medicine (J.D.), Great Ormond Street Hospital for Children, London United Kingdom; Department of Genetic Medicine (G.V.R.), Johns Hopkins, Baltimore, MD; Division of Pediatric Blood and Marrow Transplantation & Cellular Therapy (T.L., P.J.O.), University of Minnesota, Minneapolis; Pediatric Oncology (J.-S.K.), Hematology, Hemostaseology, University Hospital Leipzig, Germany; Pediatric Blood and Bone Marrow Transplantation (C.A.L.), Princess Maxima Center Utrecht, the Netherlands; Department of Pediatrics (C.A.L.), Wilhemina Children's Hospital, UMC Utrecht, Utrecht University, the Netherlands; Director of Pediatric Neuroimaging (P.C.), Lenox Hill Radiology and Medical Imaging Associates, New York, NY; Moser Center for Leukodystrophies (B.R.T., A.F.), Kennedy Krieger Institute, Johns Hopkins Medical Institutions, Baltimore, MD; Department of Neurogenetics (A.B.M.), Kennedy Krieger Institute, Baltimore, MD; Laboratory Genetic Metabolic Diseases (F.M.V., S.F., S.K.), Department of Clinical Chemistry and Pediatrics, Amsterdam UMC, Amsterdam Gastroenterology Endocrinology Metabolism, University of Amsterdam, the Netherlands; and Department of Neurology (F.S.E.), Massachusetts General Hospital, Boston, MA. Dr. van Ballegoij is currently at the Department of Neurology, Zaans Medisch Centrum, Zaandam., Caruso P; From the Department of Pediatric Neurology/Emma Children's Hospital (M.E., W.J.C.B., I.C.H.), Amsterdam UMC, Amsterdam Leukodystrophy Center, University of Amsterdam, the Netherlands; Division of Child Neurology (E.J.M.), Department of Pediatrics, Weill Cornell Medicine/NewYork-Presbyterian Hospital, NY; Department of Neurology & Pediatrics/Lucile Packard Children's Hospital (K.P.V.H.), Stanford University School of Medicine, Palo Alto, CA 4. Department of Neurology, Leukodystrophy Clinic, University of Leipzig Medical Center, Germany; Unit of Rare Neurodegenerative and Neurometabolic Diseases (E.S.), Fondazione IRCCS Istituto Neurologico C. Besta, Milano, Italy; Department of Pediatric Endocrinology/Emma Children's Hospital (A.S.P.T.), Amsterdam UMC, University of Amsterdam, the Netherlands; AP-HP (F.M.), Department of Medical Genetics, Reference Center for Adult Neurometabolic Diseases and Leukodystrophies, and INSERM U 1127, CNRS UMR 7225, Paris Brain Institute, La Pitié-Salpêtrière University Hospital, Paris, France; Department of Pediatric Neurology/Hôpital Bicêtre Paris Sud (C.S.), France, Reference Center for Children Leukodystrophies Inserm U1127, ICM-Hôpital Pitié Salpêtrière, Paris, France; Division of Pediatric Endocrinology and Diabetes (M.O.R.), Children's Hospital at Montefiore, Albert Einstein College of Medicine of Medicine, Bronx, NY; Neuroendocrine Unit (N.A.T.), Massachusetts General Hospital, Boston, MA; Harvard Medical School (N.A.T.), Boston, MA; Division of Pediatric Endocrinology (A.H.), Department of Pediatrics, Massachusetts General Hospital, Boston, MA, and Harvard Medical School (A.H.), Boston, MA; Charles Dent Metabolic Unit (R.H.L.), National Hospital for Neurology and Neurosurgery, London, United Kingdom; Metabolic Medicine (J.D.), Great Ormond Street Hospital for Children, London United Kingdom; Department of Genetic Medicine (G.V.R.), Johns Hopkins, Baltimore, MD; Division of Pediatric Blood and Marrow Transplantation & Cellular Therapy (T.L., P.J.O.), University of Minnesota, Minneapolis; Pediatric Oncology (J.-S.K.), Hematology, Hemostaseology, University Hospital Leipzig, Germany; Pediatric Blood and Bone Marrow Transplantation (C.A.L.), Princess Maxima Center Utrecht, the Netherlands; Department of Pediatrics (C.A.L.), Wilhemina Children's Hospital, UMC Utrecht, Utrecht University, the Netherlands; Director of Pediatric Neuroimaging (P.C.), Lenox Hill Radiology and Medical Imaging Associates, New York, NY; Moser Center for Leukodystrophies (B.R.T., A.F.), Kennedy Krieger Institute, Johns Hopkins Medical Institutions, Baltimore, MD; Department of Neurogenetics (A.B.M.), Kennedy Krieger Institute, Baltimore, MD; Laboratory Genetic Metabolic Diseases (F.M.V., S.F., S.K.), Department of Clinical Chemistry and Pediatrics, Amsterdam UMC, Amsterdam Gastroenterology Endocrinology Metabolism, University of Amsterdam, the Netherlands; and Department of Neurology (F.S.E.), Massachusetts General Hospital, Boston, MA. Dr. van Ballegoij is currently at the Department of Neurology, Zaans Medisch Centrum, Zaandam., Turk BR; From the Department of Pediatric Neurology/Emma Children's Hospital (M.E., W.J.C.B., I.C.H.), Amsterdam UMC, Amsterdam Leukodystrophy Center, University of Amsterdam, the Netherlands; Division of Child Neurology (E.J.M.), Department of Pediatrics, Weill Cornell Medicine/NewYork-Presbyterian Hospital, NY; Department of Neurology & Pediatrics/Lucile Packard Children's Hospital (K.P.V.H.), Stanford University School of Medicine, Palo Alto, CA 4. Department of Neurology, Leukodystrophy Clinic, University of Leipzig Medical Center, Germany; Unit of Rare Neurodegenerative and Neurometabolic Diseases (E.S.), Fondazione IRCCS Istituto Neurologico C. Besta, Milano, Italy; Department of Pediatric Endocrinology/Emma Children's Hospital (A.S.P.T.), Amsterdam UMC, University of Amsterdam, the Netherlands; AP-HP (F.M.), Department of Medical Genetics, Reference Center for Adult Neurometabolic Diseases and Leukodystrophies, and INSERM U 1127, CNRS UMR 7225, Paris Brain Institute, La Pitié-Salpêtrière University Hospital, Paris, France; Department of Pediatric Neurology/Hôpital Bicêtre Paris Sud (C.S.), France, Reference Center for Children Leukodystrophies Inserm U1127, ICM-Hôpital Pitié Salpêtrière, Paris, France; Division of Pediatric Endocrinology and Diabetes (M.O.R.), Children's Hospital at Montefiore, Albert Einstein College of Medicine of Medicine, Bronx, NY; Neuroendocrine Unit (N.A.T.), Massachusetts General Hospital, Boston, MA; Harvard Medical School (N.A.T.), Boston, MA; Division of Pediatric Endocrinology (A.H.), Department of Pediatrics, Massachusetts General Hospital, Boston, MA, and Harvard Medical School (A.H.), Boston, MA; Charles Dent Metabolic Unit (R.H.L.), National Hospital for Neurology and Neurosurgery, London, United Kingdom; Metabolic Medicine (J.D.), Great Ormond Street Hospital for Children, London United Kingdom; Department of Genetic Medicine (G.V.R.), Johns Hopkins, Baltimore, MD; Division of Pediatric Blood and Marrow Transplantation & Cellular Therapy (T.L., P.J.O.), University of Minnesota, Minneapolis; Pediatric Oncology (J.-S.K.), Hematology, Hemostaseology, University Hospital Leipzig, Germany; Pediatric Blood and Bone Marrow Transplantation (C.A.L.), Princess Maxima Center Utrecht, the Netherlands; Department of Pediatrics (C.A.L.), Wilhemina Children's Hospital, UMC Utrecht, Utrecht University, the Netherlands; Director of Pediatric Neuroimaging (P.C.), Lenox Hill Radiology and Medical Imaging Associates, New York, NY; Moser Center for Leukodystrophies (B.R.T., A.F.), Kennedy Krieger Institute, Johns Hopkins Medical Institutions, Baltimore, MD; Department of Neurogenetics (A.B.M.), Kennedy Krieger Institute, Baltimore, MD; Laboratory Genetic Metabolic Diseases (F.M.V., S.F., S.K.), Department of Clinical Chemistry and Pediatrics, Amsterdam UMC, Amsterdam Gastroenterology Endocrinology Metabolism, University of Amsterdam, the Netherlands; and Department of Neurology (F.S.E.), Massachusetts General Hospital, Boston, MA. Dr. van Ballegoij is currently at the Department of Neurology, Zaans Medisch Centrum, Zaandam., Moser AB; From the Department of Pediatric Neurology/Emma Children's Hospital (M.E., W.J.C.B., I.C.H.), Amsterdam UMC, Amsterdam Leukodystrophy Center, University of Amsterdam, the Netherlands; Division of Child Neurology (E.J.M.), Department of Pediatrics, Weill Cornell Medicine/NewYork-Presbyterian Hospital, NY; Department of Neurology & Pediatrics/Lucile Packard Children's Hospital (K.P.V.H.), Stanford University School of Medicine, Palo Alto, CA 4. Department of Neurology, Leukodystrophy Clinic, University of Leipzig Medical Center, Germany; Unit of Rare Neurodegenerative and Neurometabolic Diseases (E.S.), Fondazione IRCCS Istituto Neurologico C. Besta, Milano, Italy; Department of Pediatric Endocrinology/Emma Children's Hospital (A.S.P.T.), Amsterdam UMC, University of Amsterdam, the Netherlands; AP-HP (F.M.), Department of Medical Genetics, Reference Center for Adult Neurometabolic Diseases and Leukodystrophies, and INSERM U 1127, CNRS UMR 7225, Paris Brain Institute, La Pitié-Salpêtrière University Hospital, Paris, France; Department of Pediatric Neurology/Hôpital Bicêtre Paris Sud (C.S.), France, Reference Center for Children Leukodystrophies Inserm U1127, ICM-Hôpital Pitié Salpêtrière, Paris, France; Division of Pediatric Endocrinology and Diabetes (M.O.R.), Children's Hospital at Montefiore, Albert Einstein College of Medicine of Medicine, Bronx, NY; Neuroendocrine Unit (N.A.T.), Massachusetts General Hospital, Boston, MA; Harvard Medical School (N.A.T.), Boston, MA; Division of Pediatric Endocrinology (A.H.), Department of Pediatrics, Massachusetts General Hospital, Boston, MA, and Harvard Medical School (A.H.), Boston, MA; Charles Dent Metabolic Unit (R.H.L.), National Hospital for Neurology and Neurosurgery, London, United Kingdom; Metabolic Medicine (J.D.), Great Ormond Street Hospital for Children, London United Kingdom; Department of Genetic Medicine (G.V.R.), Johns Hopkins, Baltimore, MD; Division of Pediatric Blood and Marrow Transplantation & Cellular Therapy (T.L., P.J.O.), University of Minnesota, Minneapolis; Pediatric Oncology (J.-S.K.), Hematology, Hemostaseology, University Hospital Leipzig, Germany; Pediatric Blood and Bone Marrow Transplantation (C.A.L.), Princess Maxima Center Utrecht, the Netherlands; Department of Pediatrics (C.A.L.), Wilhemina Children's Hospital, UMC Utrecht, Utrecht University, the Netherlands; Director of Pediatric Neuroimaging (P.C.), Lenox Hill Radiology and Medical Imaging Associates, New York, NY; Moser Center for Leukodystrophies (B.R.T., A.F.), Kennedy Krieger Institute, Johns Hopkins Medical Institutions, Baltimore, MD; Department of Neurogenetics (A.B.M.), Kennedy Krieger Institute, Baltimore, MD; Laboratory Genetic Metabolic Diseases (F.M.V., S.F., S.K.), Department of Clinical Chemistry and Pediatrics, Amsterdam UMC, Amsterdam Gastroenterology Endocrinology Metabolism, University of Amsterdam, the Netherlands; and Department of Neurology (F.S.E.), Massachusetts General Hospital, Boston, MA. Dr. van Ballegoij is currently at the Department of Neurology, Zaans Medisch Centrum, Zaandam., Vaz FM; From the Department of Pediatric Neurology/Emma Children's Hospital (M.E., W.J.C.B., I.C.H.), Amsterdam UMC, Amsterdam Leukodystrophy Center, University of Amsterdam, the Netherlands; Division of Child Neurology (E.J.M.), Department of Pediatrics, Weill Cornell Medicine/NewYork-Presbyterian Hospital, NY; Department of Neurology & Pediatrics/Lucile Packard Children's Hospital (K.P.V.H.), Stanford University School of Medicine, Palo Alto, CA 4. Department of Neurology, Leukodystrophy Clinic, University of Leipzig Medical Center, Germany; Unit of Rare Neurodegenerative and Neurometabolic Diseases (E.S.), Fondazione IRCCS Istituto Neurologico C. Besta, Milano, Italy; Department of Pediatric Endocrinology/Emma Children's Hospital (A.S.P.T.), Amsterdam UMC, University of Amsterdam, the Netherlands; AP-HP (F.M.), Department of Medical Genetics, Reference Center for Adult Neurometabolic Diseases and Leukodystrophies, and INSERM U 1127, CNRS UMR 7225, Paris Brain Institute, La Pitié-Salpêtrière University Hospital, Paris, France; Department of Pediatric Neurology/Hôpital Bicêtre Paris Sud (C.S.), France, Reference Center for Children Leukodystrophies Inserm U1127, ICM-Hôpital Pitié Salpêtrière, Paris, France; Division of Pediatric Endocrinology and Diabetes (M.O.R.), Children's Hospital at Montefiore, Albert Einstein College of Medicine of Medicine, Bronx, NY; Neuroendocrine Unit (N.A.T.), Massachusetts General Hospital, Boston, MA; Harvard Medical School (N.A.T.), Boston, MA; Division of Pediatric Endocrinology (A.H.), Department of Pediatrics, Massachusetts General Hospital, Boston, MA, and Harvard Medical School (A.H.), Boston, MA; Charles Dent Metabolic Unit (R.H.L.), National Hospital for Neurology and Neurosurgery, London, United Kingdom; Metabolic Medicine (J.D.), Great Ormond Street Hospital for Children, London United Kingdom; Department of Genetic Medicine (G.V.R.), Johns Hopkins, Baltimore, MD; Division of Pediatric Blood and Marrow Transplantation & Cellular Therapy (T.L., P.J.O.), University of Minnesota, Minneapolis; Pediatric Oncology (J.-S.K.), Hematology, Hemostaseology, University Hospital Leipzig, Germany; Pediatric Blood and Bone Marrow Transplantation (C.A.L.), Princess Maxima Center Utrecht, the Netherlands; Department of Pediatrics (C.A.L.), Wilhemina Children's Hospital, UMC Utrecht, Utrecht University, the Netherlands; Director of Pediatric Neuroimaging (P.C.), Lenox Hill Radiology and Medical Imaging Associates, New York, NY; Moser Center for Leukodystrophies (B.R.T., A.F.), Kennedy Krieger Institute, Johns Hopkins Medical Institutions, Baltimore, MD; Department of Neurogenetics (A.B.M.), Kennedy Krieger Institute, Baltimore, MD; Laboratory Genetic Metabolic Diseases (F.M.V., S.F., S.K.), Department of Clinical Chemistry and Pediatrics, Amsterdam UMC, Amsterdam Gastroenterology Endocrinology Metabolism, University of Amsterdam, the Netherlands; and Department of Neurology (F.S.E.), Massachusetts General Hospital, Boston, MA. Dr. van Ballegoij is currently at the Department of Neurology, Zaans Medisch Centrum, Zaandam., Ferdinandusse S; From the Department of Pediatric Neurology/Emma Children's Hospital (M.E., W.J.C.B., I.C.H.), Amsterdam UMC, Amsterdam Leukodystrophy Center, University of Amsterdam, the Netherlands; Division of Child Neurology (E.J.M.), Department of Pediatrics, Weill Cornell Medicine/NewYork-Presbyterian Hospital, NY; Department of Neurology & Pediatrics/Lucile Packard Children's Hospital (K.P.V.H.), Stanford University School of Medicine, Palo Alto, CA 4. Department of Neurology, Leukodystrophy Clinic, University of Leipzig Medical Center, Germany; Unit of Rare Neurodegenerative and Neurometabolic Diseases (E.S.), Fondazione IRCCS Istituto Neurologico C. Besta, Milano, Italy; Department of Pediatric Endocrinology/Emma Children's Hospital (A.S.P.T.), Amsterdam UMC, University of Amsterdam, the Netherlands; AP-HP (F.M.), Department of Medical Genetics, Reference Center for Adult Neurometabolic Diseases and Leukodystrophies, and INSERM U 1127, CNRS UMR 7225, Paris Brain Institute, La Pitié-Salpêtrière University Hospital, Paris, France; Department of Pediatric Neurology/Hôpital Bicêtre Paris Sud (C.S.), France, Reference Center for Children Leukodystrophies Inserm U1127, ICM-Hôpital Pitié Salpêtrière, Paris, France; Division of Pediatric Endocrinology and Diabetes (M.O.R.), Children's Hospital at Montefiore, Albert Einstein College of Medicine of Medicine, Bronx, NY; Neuroendocrine Unit (N.A.T.), Massachusetts General Hospital, Boston, MA; Harvard Medical School (N.A.T.), Boston, MA; Division of Pediatric Endocrinology (A.H.), Department of Pediatrics, Massachusetts General Hospital, Boston, MA, and Harvard Medical School (A.H.), Boston, MA; Charles Dent Metabolic Unit (R.H.L.), National Hospital for Neurology and Neurosurgery, London, United Kingdom; Metabolic Medicine (J.D.), Great Ormond Street Hospital for Children, London United Kingdom; Department of Genetic Medicine (G.V.R.), Johns Hopkins, Baltimore, MD; Division of Pediatric Blood and Marrow Transplantation & Cellular Therapy (T.L., P.J.O.), University of Minnesota, Minneapolis; Pediatric Oncology (J.-S.K.), Hematology, Hemostaseology, University Hospital Leipzig, Germany; Pediatric Blood and Bone Marrow Transplantation (C.A.L.), Princess Maxima Center Utrecht, the Netherlands; Department of Pediatrics (C.A.L.), Wilhemina Children's Hospital, UMC Utrecht, Utrecht University, the Netherlands; Director of Pediatric Neuroimaging (P.C.), Lenox Hill Radiology and Medical Imaging Associates, New York, NY; Moser Center for Leukodystrophies (B.R.T., A.F.), Kennedy Krieger Institute, Johns Hopkins Medical Institutions, Baltimore, MD; Department of Neurogenetics (A.B.M.), Kennedy Krieger Institute, Baltimore, MD; Laboratory Genetic Metabolic Diseases (F.M.V., S.F., S.K.), Department of Clinical Chemistry and Pediatrics, Amsterdam UMC, Amsterdam Gastroenterology Endocrinology Metabolism, University of Amsterdam, the Netherlands; and Department of Neurology (F.S.E.), Massachusetts General Hospital, Boston, MA. Dr. van Ballegoij is currently at the Department of Neurology, Zaans Medisch Centrum, Zaandam., Kemp S; From the Department of Pediatric Neurology/Emma Children's Hospital (M.E., W.J.C.B., I.C.H.), Amsterdam UMC, Amsterdam Leukodystrophy Center, University of Amsterdam, the Netherlands; Division of Child Neurology (E.J.M.), Department of Pediatrics, Weill Cornell Medicine/NewYork-Presbyterian Hospital, NY; Department of Neurology & Pediatrics/Lucile Packard Children's Hospital (K.P.V.H.), Stanford University School of Medicine, Palo Alto, CA 4. Department of Neurology, Leukodystrophy Clinic, University of Leipzig Medical Center, Germany; Unit of Rare Neurodegenerative and Neurometabolic Diseases (E.S.), Fondazione IRCCS Istituto Neurologico C. Besta, Milano, Italy; Department of Pediatric Endocrinology/Emma Children's Hospital (A.S.P.T.), Amsterdam UMC, University of Amsterdam, the Netherlands; AP-HP (F.M.), Department of Medical Genetics, Reference Center for Adult Neurometabolic Diseases and Leukodystrophies, and INSERM U 1127, CNRS UMR 7225, Paris Brain Institute, La Pitié-Salpêtrière University Hospital, Paris, France; Department of Pediatric Neurology/Hôpital Bicêtre Paris Sud (C.S.), France, Reference Center for Children Leukodystrophies Inserm U1127, ICM-Hôpital Pitié Salpêtrière, Paris, France; Division of Pediatric Endocrinology and Diabetes (M.O.R.), Children's Hospital at Montefiore, Albert Einstein College of Medicine of Medicine, Bronx, NY; Neuroendocrine Unit (N.A.T.), Massachusetts General Hospital, Boston, MA; Harvard Medical School (N.A.T.), Boston, MA; Division of Pediatric Endocrinology (A.H.), Department of Pediatrics, Massachusetts General Hospital, Boston, MA, and Harvard Medical School (A.H.), Boston, MA; Charles Dent Metabolic Unit (R.H.L.), National Hospital for Neurology and Neurosurgery, London, United Kingdom; Metabolic Medicine (J.D.), Great Ormond Street Hospital for Children, London United Kingdom; Department of Genetic Medicine (G.V.R.), Johns Hopkins, Baltimore, MD; Division of Pediatric Blood and Marrow Transplantation & Cellular Therapy (T.L., P.J.O.), University of Minnesota, Minneapolis; Pediatric Oncology (J.-S.K.), Hematology, Hemostaseology, University Hospital Leipzig, Germany; Pediatric Blood and Bone Marrow Transplantation (C.A.L.), Princess Maxima Center Utrecht, the Netherlands; Department of Pediatrics (C.A.L.), Wilhemina Children's Hospital, UMC Utrecht, Utrecht University, the Netherlands; Director of Pediatric Neuroimaging (P.C.), Lenox Hill Radiology and Medical Imaging Associates, New York, NY; Moser Center for Leukodystrophies (B.R.T., A.F.), Kennedy Krieger Institute, Johns Hopkins Medical Institutions, Baltimore, MD; Department of Neurogenetics (A.B.M.), Kennedy Krieger Institute, Baltimore, MD; Laboratory Genetic Metabolic Diseases (F.M.V., S.F., S.K.), Department of Clinical Chemistry and Pediatrics, Amsterdam UMC, Amsterdam Gastroenterology Endocrinology Metabolism, University of Amsterdam, the Netherlands; and Department of Neurology (F.S.E.), Massachusetts General Hospital, Boston, MA. Dr. van Ballegoij is currently at the Department of Neurology, Zaans Medisch Centrum, Zaandam., Fatemi A; From the Department of Pediatric Neurology/Emma Children's Hospital (M.E., W.J.C.B., I.C.H.), Amsterdam UMC, Amsterdam Leukodystrophy Center, University of Amsterdam, the Netherlands; Division of Child Neurology (E.J.M.), Department of Pediatrics, Weill Cornell Medicine/NewYork-Presbyterian Hospital, NY; Department of Neurology & Pediatrics/Lucile Packard Children's Hospital (K.P.V.H.), Stanford University School of Medicine, Palo Alto, CA 4. Department of Neurology, Leukodystrophy Clinic, University of Leipzig Medical Center, Germany; Unit of Rare Neurodegenerative and Neurometabolic Diseases (E.S.), Fondazione IRCCS Istituto Neurologico C. Besta, Milano, Italy; Department of Pediatric Endocrinology/Emma Children's Hospital (A.S.P.T.), Amsterdam UMC, University of Amsterdam, the Netherlands; AP-HP (F.M.), Department of Medical Genetics, Reference Center for Adult Neurometabolic Diseases and Leukodystrophies, and INSERM U 1127, CNRS UMR 7225, Paris Brain Institute, La Pitié-Salpêtrière University Hospital, Paris, France; Department of Pediatric Neurology/Hôpital Bicêtre Paris Sud (C.S.), France, Reference Center for Children Leukodystrophies Inserm U1127, ICM-Hôpital Pitié Salpêtrière, Paris, France; Division of Pediatric Endocrinology and Diabetes (M.O.R.), Children's Hospital at Montefiore, Albert Einstein College of Medicine of Medicine, Bronx, NY; Neuroendocrine Unit (N.A.T.), Massachusetts General Hospital, Boston, MA; Harvard Medical School (N.A.T.), Boston, MA; Division of Pediatric Endocrinology (A.H.), Department of Pediatrics, Massachusetts General Hospital, Boston, MA, and Harvard Medical School (A.H.), Boston, MA; Charles Dent Metabolic Unit (R.H.L.), National Hospital for Neurology and Neurosurgery, London, United Kingdom; Metabolic Medicine (J.D.), Great Ormond Street Hospital for Children, London United Kingdom; Department of Genetic Medicine (G.V.R.), Johns Hopkins, Baltimore, MD; Division of Pediatric Blood and Marrow Transplantation & Cellular Therapy (T.L., P.J.O.), University of Minnesota, Minneapolis; Pediatric Oncology (J.-S.K.), Hematology, Hemostaseology, University Hospital Leipzig, Germany; Pediatric Blood and Bone Marrow Transplantation (C.A.L.), Princess Maxima Center Utrecht, the Netherlands; Department of Pediatrics (C.A.L.), Wilhemina Children's Hospital, UMC Utrecht, Utrecht University, the Netherlands; Director of Pediatric Neuroimaging (P.C.), Lenox Hill Radiology and Medical Imaging Associates, New York, NY; Moser Center for Leukodystrophies (B.R.T., A.F.), Kennedy Krieger Institute, Johns Hopkins Medical Institutions, Baltimore, MD; Department of Neurogenetics (A.B.M.), Kennedy Krieger Institute, Baltimore, MD; Laboratory Genetic Metabolic Diseases (F.M.V., S.F., S.K.), Department of Clinical Chemistry and Pediatrics, Amsterdam UMC, Amsterdam Gastroenterology Endocrinology Metabolism, University of Amsterdam, the Netherlands; and Department of Neurology (F.S.E.), Massachusetts General Hospital, Boston, MA. Dr. van Ballegoij is currently at the Department of Neurology, Zaans Medisch Centrum, Zaandam., Eichler FS; From the Department of Pediatric Neurology/Emma Children's Hospital (M.E., W.J.C.B., I.C.H.), Amsterdam UMC, Amsterdam Leukodystrophy Center, University of Amsterdam, the Netherlands; Division of Child Neurology (E.J.M.), Department of Pediatrics, Weill Cornell Medicine/NewYork-Presbyterian Hospital, NY; Department of Neurology & Pediatrics/Lucile Packard Children's Hospital (K.P.V.H.), Stanford University School of Medicine, Palo Alto, CA 4. Department of Neurology, Leukodystrophy Clinic, University of Leipzig Medical Center, Germany; Unit of Rare Neurodegenerative and Neurometabolic Diseases (E.S.), Fondazione IRCCS Istituto Neurologico C. Besta, Milano, Italy; Department of Pediatric Endocrinology/Emma Children's Hospital (A.S.P.T.), Amsterdam UMC, University of Amsterdam, the Netherlands; AP-HP (F.M.), Department of Medical Genetics, Reference Center for Adult Neurometabolic Diseases and Leukodystrophies, and INSERM U 1127, CNRS UMR 7225, Paris Brain Institute, La Pitié-Salpêtrière University Hospital, Paris, France; Department of Pediatric Neurology/Hôpital Bicêtre Paris Sud (C.S.), France, Reference Center for Children Leukodystrophies Inserm U1127, ICM-Hôpital Pitié Salpêtrière, Paris, France; Division of Pediatric Endocrinology and Diabetes (M.O.R.), Children's Hospital at Montefiore, Albert Einstein College of Medicine of Medicine, Bronx, NY; Neuroendocrine Unit (N.A.T.), Massachusetts General Hospital, Boston, MA; Harvard Medical School (N.A.T.), Boston, MA; Division of Pediatric Endocrinology (A.H.), Department of Pediatrics, Massachusetts General Hospital, Boston, MA, and Harvard Medical School (A.H.), Boston, MA; Charles Dent Metabolic Unit (R.H.L.), National Hospital for Neurology and Neurosurgery, London, United Kingdom; Metabolic Medicine (J.D.), Great Ormond Street Hospital for Children, London United Kingdom; Department of Genetic Medicine (G.V.R.), Johns Hopkins, Baltimore, MD; Division of Pediatric Blood and Marrow Transplantation & Cellular Therapy (T.L., P.J.O.), University of Minnesota, Minneapolis; Pediatric Oncology (J.-S.K.), Hematology, Hemostaseology, University Hospital Leipzig, Germany; Pediatric Blood and Bone Marrow Transplantation (C.A.L.), Princess Maxima Center Utrecht, the Netherlands; Department of Pediatrics (C.A.L.), Wilhemina Children's Hospital, UMC Utrecht, Utrecht University, the Netherlands; Director of Pediatric Neuroimaging (P.C.), Lenox Hill Radiology and Medical Imaging Associates, New York, NY; Moser Center for Leukodystrophies (B.R.T., A.F.), Kennedy Krieger Institute, Johns Hopkins Medical Institutions, Baltimore, MD; Department of Neurogenetics (A.B.M.), Kennedy Krieger Institute, Baltimore, MD; Laboratory Genetic Metabolic Diseases (F.M.V., S.F., S.K.), Department of Clinical Chemistry and Pediatrics, Amsterdam UMC, Amsterdam Gastroenterology Endocrinology Metabolism, University of Amsterdam, the Netherlands; and Department of Neurology (F.S.E.), Massachusetts General Hospital, Boston, MA. Dr. van Ballegoij is currently at the Department of Neurology, Zaans Medisch Centrum, Zaandam., Huffnagel IC; From the Department of Pediatric Neurology/Emma Children's Hospital (M.E., W.J.C.B., I.C.H.), Amsterdam UMC, Amsterdam Leukodystrophy Center, University of Amsterdam, the Netherlands; Division of Child Neurology (E.J.M.), Department of Pediatrics, Weill Cornell Medicine/NewYork-Presbyterian Hospital, NY; Department of Neurology & Pediatrics/Lucile Packard Children's Hospital (K.P.V.H.), Stanford University School of Medicine, Palo Alto, CA 4. Department of Neurology, Leukodystrophy Clinic, University of Leipzig Medical Center, Germany; Unit of Rare Neurodegenerative and Neurometabolic Diseases (E.S.), Fondazione IRCCS Istituto Neurologico C. Besta, Milano, Italy; Department of Pediatric Endocrinology/Emma Children's Hospital (A.S.P.T.), Amsterdam UMC, University of Amsterdam, the Netherlands; AP-HP (F.M.), Department of Medical Genetics, Reference Center for Adult Neurometabolic Diseases and Leukodystrophies, and INSERM U 1127, CNRS UMR 7225, Paris Brain Institute, La Pitié-Salpêtrière University Hospital, Paris, France; Department of Pediatric Neurology/Hôpital Bicêtre Paris Sud (C.S.), France, Reference Center for Children Leukodystrophies Inserm U1127, ICM-Hôpital Pitié Salpêtrière, Paris, France; Division of Pediatric Endocrinology and Diabetes (M.O.R.), Children's Hospital at Montefiore, Albert Einstein College of Medicine of Medicine, Bronx, NY; Neuroendocrine Unit (N.A.T.), Massachusetts General Hospital, Boston, MA; Harvard Medical School (N.A.T.), Boston, MA; Division of Pediatric Endocrinology (A.H.), Department of Pediatrics, Massachusetts General Hospital, Boston, MA, and Harvard Medical School (A.H.), Boston, MA; Charles Dent Metabolic Unit (R.H.L.), National Hospital for Neurology and Neurosurgery, London, United Kingdom; Metabolic Medicine (J.D.), Great Ormond Street Hospital for Children, London United Kingdom; Department of Genetic Medicine (G.V.R.), Johns Hopkins, Baltimore, MD; Division of Pediatric Blood and Marrow Transplantation & Cellular Therapy (T.L., P.J.O.), University of Minnesota, Minneapolis; Pediatric Oncology (J.-S.K.), Hematology, Hemostaseology, University Hospital Leipzig, Germany; Pediatric Blood and Bone Marrow Transplantation (C.A.L.), Princess Maxima Center Utrecht, the Netherlands; Department of Pediatrics (C.A.L.), Wilhemina Children's Hospital, UMC Utrecht, Utrecht University, the Netherlands; Director of Pediatric Neuroimaging (P.C.), Lenox Hill Radiology and Medical Imaging Associates, New York, NY; Moser Center for Leukodystrophies (B.R.T., A.F.), Kennedy Krieger Institute, Johns Hopkins Medical Institutions, Baltimore, MD; Department of Neurogenetics (A.B.M.), Kennedy Krieger Institute, Baltimore, MD; Laboratory Genetic Metabolic Diseases (F.M.V., S.F., S.K.), Department of Clinical Chemistry and Pediatrics, Amsterdam UMC, Amsterdam Gastroenterology Endocrinology Metabolism, University of Amsterdam, the Netherlands; and Department of Neurology (F.S.E.), Massachusetts General Hospital, Boston, MA. Dr. van Ballegoij is currently at the Department of Neurology, Zaans Medisch Centrum, Zaandam.
Publikováno v:
Neurology [Neurology] 2022 Nov 22; Vol. 99 (21), pp. 940-951. Date of Electronic Publication: 2022 Sep 29.
Autor:
Mallack EJ; From the Department of Pediatrics (E.J.M., A.T.), Division of Child Neurology, Weill Cornell Medical College, New York-Presbyterian Hospital; Department of Pediatrics (E.J.M.), Memorial Sloan Kettering Cancer Center, New York, NY; Department of Neurology (K.P.V.H.), Stanford University Schoolds of Medicine, Lucile Packard Children's Hospital, CA; Department of Pediatric Neurology, Emma Children's Hospital, Amsterdam University Medical Centers, the Netherlands; Department of Genetic Medicine (G.V.R.), Johns Hopkins University, Baltimore, MD; The Moser Center for Leukodystrophies (A.F.), Kennedy Krieger Institute, Johns Hopkins University, Baltimore, MD; and Department of Neurology (F.S.E.), Harvard Medical School, Massachusetts General Hospital, Boston. ejm9009@med.cornell.edu., Van Haren KP; From the Department of Pediatrics (E.J.M., A.T.), Division of Child Neurology, Weill Cornell Medical College, New York-Presbyterian Hospital; Department of Pediatrics (E.J.M.), Memorial Sloan Kettering Cancer Center, New York, NY; Department of Neurology (K.P.V.H.), Stanford University Schoolds of Medicine, Lucile Packard Children's Hospital, CA; Department of Pediatric Neurology, Emma Children's Hospital, Amsterdam University Medical Centers, the Netherlands; Department of Genetic Medicine (G.V.R.), Johns Hopkins University, Baltimore, MD; The Moser Center for Leukodystrophies (A.F.), Kennedy Krieger Institute, Johns Hopkins University, Baltimore, MD; and Department of Neurology (F.S.E.), Harvard Medical School, Massachusetts General Hospital, Boston., Torrey A; From the Department of Pediatrics (E.J.M., A.T.), Division of Child Neurology, Weill Cornell Medical College, New York-Presbyterian Hospital; Department of Pediatrics (E.J.M.), Memorial Sloan Kettering Cancer Center, New York, NY; Department of Neurology (K.P.V.H.), Stanford University Schoolds of Medicine, Lucile Packard Children's Hospital, CA; Department of Pediatric Neurology, Emma Children's Hospital, Amsterdam University Medical Centers, the Netherlands; Department of Genetic Medicine (G.V.R.), Johns Hopkins University, Baltimore, MD; The Moser Center for Leukodystrophies (A.F.), Kennedy Krieger Institute, Johns Hopkins University, Baltimore, MD; and Department of Neurology (F.S.E.), Harvard Medical School, Massachusetts General Hospital, Boston., van de Stadt S; From the Department of Pediatrics (E.J.M., A.T.), Division of Child Neurology, Weill Cornell Medical College, New York-Presbyterian Hospital; Department of Pediatrics (E.J.M.), Memorial Sloan Kettering Cancer Center, New York, NY; Department of Neurology (K.P.V.H.), Stanford University Schoolds of Medicine, Lucile Packard Children's Hospital, CA; Department of Pediatric Neurology, Emma Children's Hospital, Amsterdam University Medical Centers, the Netherlands; Department of Genetic Medicine (G.V.R.), Johns Hopkins University, Baltimore, MD; The Moser Center for Leukodystrophies (A.F.), Kennedy Krieger Institute, Johns Hopkins University, Baltimore, MD; and Department of Neurology (F.S.E.), Harvard Medical School, Massachusetts General Hospital, Boston., Engelen M; From the Department of Pediatrics (E.J.M., A.T.), Division of Child Neurology, Weill Cornell Medical College, New York-Presbyterian Hospital; Department of Pediatrics (E.J.M.), Memorial Sloan Kettering Cancer Center, New York, NY; Department of Neurology (K.P.V.H.), Stanford University Schoolds of Medicine, Lucile Packard Children's Hospital, CA; Department of Pediatric Neurology, Emma Children's Hospital, Amsterdam University Medical Centers, the Netherlands; Department of Genetic Medicine (G.V.R.), Johns Hopkins University, Baltimore, MD; The Moser Center for Leukodystrophies (A.F.), Kennedy Krieger Institute, Johns Hopkins University, Baltimore, MD; and Department of Neurology (F.S.E.), Harvard Medical School, Massachusetts General Hospital, Boston., Raymond GV; From the Department of Pediatrics (E.J.M., A.T.), Division of Child Neurology, Weill Cornell Medical College, New York-Presbyterian Hospital; Department of Pediatrics (E.J.M.), Memorial Sloan Kettering Cancer Center, New York, NY; Department of Neurology (K.P.V.H.), Stanford University Schoolds of Medicine, Lucile Packard Children's Hospital, CA; Department of Pediatric Neurology, Emma Children's Hospital, Amsterdam University Medical Centers, the Netherlands; Department of Genetic Medicine (G.V.R.), Johns Hopkins University, Baltimore, MD; The Moser Center for Leukodystrophies (A.F.), Kennedy Krieger Institute, Johns Hopkins University, Baltimore, MD; and Department of Neurology (F.S.E.), Harvard Medical School, Massachusetts General Hospital, Boston., Fatemi A; From the Department of Pediatrics (E.J.M., A.T.), Division of Child Neurology, Weill Cornell Medical College, New York-Presbyterian Hospital; Department of Pediatrics (E.J.M.), Memorial Sloan Kettering Cancer Center, New York, NY; Department of Neurology (K.P.V.H.), Stanford University Schoolds of Medicine, Lucile Packard Children's Hospital, CA; Department of Pediatric Neurology, Emma Children's Hospital, Amsterdam University Medical Centers, the Netherlands; Department of Genetic Medicine (G.V.R.), Johns Hopkins University, Baltimore, MD; The Moser Center for Leukodystrophies (A.F.), Kennedy Krieger Institute, Johns Hopkins University, Baltimore, MD; and Department of Neurology (F.S.E.), Harvard Medical School, Massachusetts General Hospital, Boston., Eichler FS; From the Department of Pediatrics (E.J.M., A.T.), Division of Child Neurology, Weill Cornell Medical College, New York-Presbyterian Hospital; Department of Pediatrics (E.J.M.), Memorial Sloan Kettering Cancer Center, New York, NY; Department of Neurology (K.P.V.H.), Stanford University Schoolds of Medicine, Lucile Packard Children's Hospital, CA; Department of Pediatric Neurology, Emma Children's Hospital, Amsterdam University Medical Centers, the Netherlands; Department of Genetic Medicine (G.V.R.), Johns Hopkins University, Baltimore, MD; The Moser Center for Leukodystrophies (A.F.), Kennedy Krieger Institute, Johns Hopkins University, Baltimore, MD; and Department of Neurology (F.S.E.), Harvard Medical School, Massachusetts General Hospital, Boston.
Publikováno v:
Neurology [Neurology] 2022 Aug 01; Vol. 99 (5), pp. e512-e520. Date of Electronic Publication: 2022 Aug 01.
Autor:
Santoro JD; Division of Child Neurology, Lucile Packard Children's Hospital at Stanford, Palo Alto, CA, USA. jsantoro@stanford.edu., Lazzareschi DV; Stanford University School of Medicine, Palo Alto, CA, USA., Campen CJ; Division of Child Neurology, Lucile Packard Children's Hospital at Stanford, Palo Alto, CA, USA., Van Haren KP; Division of Child Neurology, Lucile Packard Children's Hospital at Stanford, Palo Alto, CA, USA.
Publikováno v:
Journal of neurology [J Neurol] 2018 Jan; Vol. 265 (1), pp. 141-150. Date of Electronic Publication: 2017 Nov 24.