Zobrazeno 1 - 10
of 26
pro vyhledávání: '"Bharucha-Goebel DX"'
Autor:
Storch, K, Spuler, S, Day, JW, Jones, KJ, Bharucha-Goebel, DX, Salort Campana, E, Walter, MC, Krause, S, Pestronk, A, Paradas, C, Stojkovic, T, Mori-Yoshimura, M, Bravver, E, Manera, JD, Pegoraro, E, Mendell, JR, Bushby, K, Straub, V
Publikováno v:
Nervenheilkunde; May 2019, Vol. 38 Issue: 5
Autor:
Straub, V, James, MK, Mayhew, AG, Spuler, S, Day, JW, Jones, KJ, Bharucha-Goebel, DX, Salort Campana, E, Pestronk, A, Krause, S, Walter, MC, Paradas, C, Stojkovic, T, Yoshimura, M, Bravver, E, Díaz-Manera, J, Pegoraro, E, Mendell, J, Bushby, K
Publikováno v:
Nervenheilkunde; May 2019, Vol. 38 Issue: 5
Autor:
Foley AR; From the Neuromuscular and Neurogenetics Disorders of Childhood Section (A.R.F., P.Y., M.E.L., S.B.N., G.V.A., Y.H., L.H.H., S.D., D.X.B.-G., Y.Z., M.F., J.D., C.M., C.A., C.G.B.), Neurogenetics Branch, NINDS, NIH, Bethesda, MD; Division of Neurology (M.E.L.), Oregon Health and Science University, Portland, OR; Department of Neurology (L.H.H.), Boston Children's Hospital, MA; Rehabilitation Medicine Department (M.S.J., M.W.); Occupational Therapy Section (R.P.), Rehabilitation Medicine Department, NIH, Bethesda, MD; Division of Neurology (D.X.B.-G.), Children's National Hospital, Washington, DC; Division of Pulmonology (O.M.), Children's Hospital of Philadelphia, PA; Santhera Pharmaceuticals (R.H., D.P.), Pratteln, Switzerland; and Department of Biostatistics (K.C.), Mailman School of Public Health, Columbia University, NY., Yun P; From the Neuromuscular and Neurogenetics Disorders of Childhood Section (A.R.F., P.Y., M.E.L., S.B.N., G.V.A., Y.H., L.H.H., S.D., D.X.B.-G., Y.Z., M.F., J.D., C.M., C.A., C.G.B.), Neurogenetics Branch, NINDS, NIH, Bethesda, MD; Division of Neurology (M.E.L.), Oregon Health and Science University, Portland, OR; Department of Neurology (L.H.H.), Boston Children's Hospital, MA; Rehabilitation Medicine Department (M.S.J., M.W.); Occupational Therapy Section (R.P.), Rehabilitation Medicine Department, NIH, Bethesda, MD; Division of Neurology (D.X.B.-G.), Children's National Hospital, Washington, DC; Division of Pulmonology (O.M.), Children's Hospital of Philadelphia, PA; Santhera Pharmaceuticals (R.H., D.P.), Pratteln, Switzerland; and Department of Biostatistics (K.C.), Mailman School of Public Health, Columbia University, NY., Leach ME; From the Neuromuscular and Neurogenetics Disorders of Childhood Section (A.R.F., P.Y., M.E.L., S.B.N., G.V.A., Y.H., L.H.H., S.D., D.X.B.-G., Y.Z., M.F., J.D., C.M., C.A., C.G.B.), Neurogenetics Branch, NINDS, NIH, Bethesda, MD; Division of Neurology (M.E.L.), Oregon Health and Science University, Portland, OR; Department of Neurology (L.H.H.), Boston Children's Hospital, MA; Rehabilitation Medicine Department (M.S.J., M.W.); Occupational Therapy Section (R.P.), Rehabilitation Medicine Department, NIH, Bethesda, MD; Division of Neurology (D.X.B.-G.), Children's National Hospital, Washington, DC; Division of Pulmonology (O.M.), Children's Hospital of Philadelphia, PA; Santhera Pharmaceuticals (R.H., D.P.), Pratteln, Switzerland; and Department of Biostatistics (K.C.), Mailman School of Public Health, Columbia University, NY., Neuhaus SB; From the Neuromuscular and Neurogenetics Disorders of Childhood Section (A.R.F., P.Y., M.E.L., S.B.N., G.V.A., Y.H., L.H.H., S.D., D.X.B.-G., Y.Z., M.F., J.D., C.M., C.A., C.G.B.), Neurogenetics Branch, NINDS, NIH, Bethesda, MD; Division of Neurology (M.E.L.), Oregon Health and Science University, Portland, OR; Department of Neurology (L.H.H.), Boston Children's Hospital, MA; Rehabilitation Medicine Department (M.S.J., M.W.); Occupational Therapy Section (R.P.), Rehabilitation Medicine Department, NIH, Bethesda, MD; Division of Neurology (D.X.B.-G.), Children's National Hospital, Washington, DC; Division of Pulmonology (O.M.), Children's Hospital of Philadelphia, PA; Santhera Pharmaceuticals (R.H., D.P.), Pratteln, Switzerland; and Department of Biostatistics (K.C.), Mailman School of Public Health, Columbia University, NY., Averion GV; From the Neuromuscular and Neurogenetics Disorders of Childhood Section (A.R.F., P.Y., M.E.L., S.B.N., G.V.A., Y.H., L.H.H., S.D., D.X.B.-G., Y.Z., M.F., J.D., C.M., C.A., C.G.B.), Neurogenetics Branch, NINDS, NIH, Bethesda, MD; Division of Neurology (M.E.L.), Oregon Health and Science University, Portland, OR; Department of Neurology (L.H.H.), Boston Children's Hospital, MA; Rehabilitation Medicine Department (M.S.J., M.W.); Occupational Therapy Section (R.P.), Rehabilitation Medicine Department, NIH, Bethesda, MD; Division of Neurology (D.X.B.-G.), Children's National Hospital, Washington, DC; Division of Pulmonology (O.M.), Children's Hospital of Philadelphia, PA; Santhera Pharmaceuticals (R.H., D.P.), Pratteln, Switzerland; and Department of Biostatistics (K.C.), Mailman School of Public Health, Columbia University, NY., Hu Y; From the Neuromuscular and Neurogenetics Disorders of Childhood Section (A.R.F., P.Y., M.E.L., S.B.N., G.V.A., Y.H., L.H.H., S.D., D.X.B.-G., Y.Z., M.F., J.D., C.M., C.A., C.G.B.), Neurogenetics Branch, NINDS, NIH, Bethesda, MD; Division of Neurology (M.E.L.), Oregon Health and Science University, Portland, OR; Department of Neurology (L.H.H.), Boston Children's Hospital, MA; Rehabilitation Medicine Department (M.S.J., M.W.); Occupational Therapy Section (R.P.), Rehabilitation Medicine Department, NIH, Bethesda, MD; Division of Neurology (D.X.B.-G.), Children's National Hospital, Washington, DC; Division of Pulmonology (O.M.), Children's Hospital of Philadelphia, PA; Santhera Pharmaceuticals (R.H., D.P.), Pratteln, Switzerland; and Department of Biostatistics (K.C.), Mailman School of Public Health, Columbia University, NY., Hayes LH; From the Neuromuscular and Neurogenetics Disorders of Childhood Section (A.R.F., P.Y., M.E.L., S.B.N., G.V.A., Y.H., L.H.H., S.D., D.X.B.-G., Y.Z., M.F., J.D., C.M., C.A., C.G.B.), Neurogenetics Branch, NINDS, NIH, Bethesda, MD; Division of Neurology (M.E.L.), Oregon Health and Science University, Portland, OR; Department of Neurology (L.H.H.), Boston Children's Hospital, MA; Rehabilitation Medicine Department (M.S.J., M.W.); Occupational Therapy Section (R.P.), Rehabilitation Medicine Department, NIH, Bethesda, MD; Division of Neurology (D.X.B.-G.), Children's National Hospital, Washington, DC; Division of Pulmonology (O.M.), Children's Hospital of Philadelphia, PA; Santhera Pharmaceuticals (R.H., D.P.), Pratteln, Switzerland; and Department of Biostatistics (K.C.), Mailman School of Public Health, Columbia University, NY., Donkervoort S; From the Neuromuscular and Neurogenetics Disorders of Childhood Section (A.R.F., P.Y., M.E.L., S.B.N., G.V.A., Y.H., L.H.H., S.D., D.X.B.-G., Y.Z., M.F., J.D., C.M., C.A., C.G.B.), Neurogenetics Branch, NINDS, NIH, Bethesda, MD; Division of Neurology (M.E.L.), Oregon Health and Science University, Portland, OR; Department of Neurology (L.H.H.), Boston Children's Hospital, MA; Rehabilitation Medicine Department (M.S.J., M.W.); Occupational Therapy Section (R.P.), Rehabilitation Medicine Department, NIH, Bethesda, MD; Division of Neurology (D.X.B.-G.), Children's National Hospital, Washington, DC; Division of Pulmonology (O.M.), Children's Hospital of Philadelphia, PA; Santhera Pharmaceuticals (R.H., D.P.), Pratteln, Switzerland; and Department of Biostatistics (K.C.), Mailman School of Public Health, Columbia University, NY., Jain MS; From the Neuromuscular and Neurogenetics Disorders of Childhood Section (A.R.F., P.Y., M.E.L., S.B.N., G.V.A., Y.H., L.H.H., S.D., D.X.B.-G., Y.Z., M.F., J.D., C.M., C.A., C.G.B.), Neurogenetics Branch, NINDS, NIH, Bethesda, MD; Division of Neurology (M.E.L.), Oregon Health and Science University, Portland, OR; Department of Neurology (L.H.H.), Boston Children's Hospital, MA; Rehabilitation Medicine Department (M.S.J., M.W.); Occupational Therapy Section (R.P.), Rehabilitation Medicine Department, NIH, Bethesda, MD; Division of Neurology (D.X.B.-G.), Children's National Hospital, Washington, DC; Division of Pulmonology (O.M.), Children's Hospital of Philadelphia, PA; Santhera Pharmaceuticals (R.H., D.P.), Pratteln, Switzerland; and Department of Biostatistics (K.C.), Mailman School of Public Health, Columbia University, NY., Waite M; From the Neuromuscular and Neurogenetics Disorders of Childhood Section (A.R.F., P.Y., M.E.L., S.B.N., G.V.A., Y.H., L.H.H., S.D., D.X.B.-G., Y.Z., M.F., J.D., C.M., C.A., C.G.B.), Neurogenetics Branch, NINDS, NIH, Bethesda, MD; Division of Neurology (M.E.L.), Oregon Health and Science University, Portland, OR; Department of Neurology (L.H.H.), Boston Children's Hospital, MA; Rehabilitation Medicine Department (M.S.J., M.W.); Occupational Therapy Section (R.P.), Rehabilitation Medicine Department, NIH, Bethesda, MD; Division of Neurology (D.X.B.-G.), Children's National Hospital, Washington, DC; Division of Pulmonology (O.M.), Children's Hospital of Philadelphia, PA; Santhera Pharmaceuticals (R.H., D.P.), Pratteln, Switzerland; and Department of Biostatistics (K.C.), Mailman School of Public Health, Columbia University, NY., Parks R; From the Neuromuscular and Neurogenetics Disorders of Childhood Section (A.R.F., P.Y., M.E.L., S.B.N., G.V.A., Y.H., L.H.H., S.D., D.X.B.-G., Y.Z., M.F., J.D., C.M., C.A., C.G.B.), Neurogenetics Branch, NINDS, NIH, Bethesda, MD; Division of Neurology (M.E.L.), Oregon Health and Science University, Portland, OR; Department of Neurology (L.H.H.), Boston Children's Hospital, MA; Rehabilitation Medicine Department (M.S.J., M.W.); Occupational Therapy Section (R.P.), Rehabilitation Medicine Department, NIH, Bethesda, MD; Division of Neurology (D.X.B.-G.), Children's National Hospital, Washington, DC; Division of Pulmonology (O.M.), Children's Hospital of Philadelphia, PA; Santhera Pharmaceuticals (R.H., D.P.), Pratteln, Switzerland; and Department of Biostatistics (K.C.), Mailman School of Public Health, Columbia University, NY., Bharucha-Goebel DX; From the Neuromuscular and Neurogenetics Disorders of Childhood Section (A.R.F., P.Y., M.E.L., S.B.N., G.V.A., Y.H., L.H.H., S.D., D.X.B.-G., Y.Z., M.F., J.D., C.M., C.A., C.G.B.), Neurogenetics Branch, NINDS, NIH, Bethesda, MD; Division of Neurology (M.E.L.), Oregon Health and Science University, Portland, OR; Department of Neurology (L.H.H.), Boston Children's Hospital, MA; Rehabilitation Medicine Department (M.S.J., M.W.); Occupational Therapy Section (R.P.), Rehabilitation Medicine Department, NIH, Bethesda, MD; Division of Neurology (D.X.B.-G.), Children's National Hospital, Washington, DC; Division of Pulmonology (O.M.), Children's Hospital of Philadelphia, PA; Santhera Pharmaceuticals (R.H., D.P.), Pratteln, Switzerland; and Department of Biostatistics (K.C.), Mailman School of Public Health, Columbia University, NY., Mayer OH; From the Neuromuscular and Neurogenetics Disorders of Childhood Section (A.R.F., P.Y., M.E.L., S.B.N., G.V.A., Y.H., L.H.H., S.D., D.X.B.-G., Y.Z., M.F., J.D., C.M., C.A., C.G.B.), Neurogenetics Branch, NINDS, NIH, Bethesda, MD; Division of Neurology (M.E.L.), Oregon Health and Science University, Portland, OR; Department of Neurology (L.H.H.), Boston Children's Hospital, MA; Rehabilitation Medicine Department (M.S.J., M.W.); Occupational Therapy Section (R.P.), Rehabilitation Medicine Department, NIH, Bethesda, MD; Division of Neurology (D.X.B.-G.), Children's National Hospital, Washington, DC; Division of Pulmonology (O.M.), Children's Hospital of Philadelphia, PA; Santhera Pharmaceuticals (R.H., D.P.), Pratteln, Switzerland; and Department of Biostatistics (K.C.), Mailman School of Public Health, Columbia University, NY., Zou Y; From the Neuromuscular and Neurogenetics Disorders of Childhood Section (A.R.F., P.Y., M.E.L., S.B.N., G.V.A., Y.H., L.H.H., S.D., D.X.B.-G., Y.Z., M.F., J.D., C.M., C.A., C.G.B.), Neurogenetics Branch, NINDS, NIH, Bethesda, MD; Division of Neurology (M.E.L.), Oregon Health and Science University, Portland, OR; Department of Neurology (L.H.H.), Boston Children's Hospital, MA; Rehabilitation Medicine Department (M.S.J., M.W.); Occupational Therapy Section (R.P.), Rehabilitation Medicine Department, NIH, Bethesda, MD; Division of Neurology (D.X.B.-G.), Children's National Hospital, Washington, DC; Division of Pulmonology (O.M.), Children's Hospital of Philadelphia, PA; Santhera Pharmaceuticals (R.H., D.P.), Pratteln, Switzerland; and Department of Biostatistics (K.C.), Mailman School of Public Health, Columbia University, NY., Fink M; From the Neuromuscular and Neurogenetics Disorders of Childhood Section (A.R.F., P.Y., M.E.L., S.B.N., G.V.A., Y.H., L.H.H., S.D., D.X.B.-G., Y.Z., M.F., J.D., C.M., C.A., C.G.B.), Neurogenetics Branch, NINDS, NIH, Bethesda, MD; Division of Neurology (M.E.L.), Oregon Health and Science University, Portland, OR; Department of Neurology (L.H.H.), Boston Children's Hospital, MA; Rehabilitation Medicine Department (M.S.J., M.W.); Occupational Therapy Section (R.P.), Rehabilitation Medicine Department, NIH, Bethesda, MD; Division of Neurology (D.X.B.-G.), Children's National Hospital, Washington, DC; Division of Pulmonology (O.M.), Children's Hospital of Philadelphia, PA; Santhera Pharmaceuticals (R.H., D.P.), Pratteln, Switzerland; and Department of Biostatistics (K.C.), Mailman School of Public Health, Columbia University, NY., DeCoster J; From the Neuromuscular and Neurogenetics Disorders of Childhood Section (A.R.F., P.Y., M.E.L., S.B.N., G.V.A., Y.H., L.H.H., S.D., D.X.B.-G., Y.Z., M.F., J.D., C.M., C.A., C.G.B.), Neurogenetics Branch, NINDS, NIH, Bethesda, MD; Division of Neurology (M.E.L.), Oregon Health and Science University, Portland, OR; Department of Neurology (L.H.H.), Boston Children's Hospital, MA; Rehabilitation Medicine Department (M.S.J., M.W.); Occupational Therapy Section (R.P.), Rehabilitation Medicine Department, NIH, Bethesda, MD; Division of Neurology (D.X.B.-G.), Children's National Hospital, Washington, DC; Division of Pulmonology (O.M.), Children's Hospital of Philadelphia, PA; Santhera Pharmaceuticals (R.H., D.P.), Pratteln, Switzerland; and Department of Biostatistics (K.C.), Mailman School of Public Health, Columbia University, NY., Mendoza C; From the Neuromuscular and Neurogenetics Disorders of Childhood Section (A.R.F., P.Y., M.E.L., S.B.N., G.V.A., Y.H., L.H.H., S.D., D.X.B.-G., Y.Z., M.F., J.D., C.M., C.A., C.G.B.), Neurogenetics Branch, NINDS, NIH, Bethesda, MD; Division of Neurology (M.E.L.), Oregon Health and Science University, Portland, OR; Department of Neurology (L.H.H.), Boston Children's Hospital, MA; Rehabilitation Medicine Department (M.S.J., M.W.); Occupational Therapy Section (R.P.), Rehabilitation Medicine Department, NIH, Bethesda, MD; Division of Neurology (D.X.B.-G.), Children's National Hospital, Washington, DC; Division of Pulmonology (O.M.), Children's Hospital of Philadelphia, PA; Santhera Pharmaceuticals (R.H., D.P.), Pratteln, Switzerland; and Department of Biostatistics (K.C.), Mailman School of Public Health, Columbia University, NY., Arévalo C; From the Neuromuscular and Neurogenetics Disorders of Childhood Section (A.R.F., P.Y., M.E.L., S.B.N., G.V.A., Y.H., L.H.H., S.D., D.X.B.-G., Y.Z., M.F., J.D., C.M., C.A., C.G.B.), Neurogenetics Branch, NINDS, NIH, Bethesda, MD; Division of Neurology (M.E.L.), Oregon Health and Science University, Portland, OR; Department of Neurology (L.H.H.), Boston Children's Hospital, MA; Rehabilitation Medicine Department (M.S.J., M.W.); Occupational Therapy Section (R.P.), Rehabilitation Medicine Department, NIH, Bethesda, MD; Division of Neurology (D.X.B.-G.), Children's National Hospital, Washington, DC; Division of Pulmonology (O.M.), Children's Hospital of Philadelphia, PA; Santhera Pharmaceuticals (R.H., D.P.), Pratteln, Switzerland; and Department of Biostatistics (K.C.), Mailman School of Public Health, Columbia University, NY., Hausmann R; From the Neuromuscular and Neurogenetics Disorders of Childhood Section (A.R.F., P.Y., M.E.L., S.B.N., G.V.A., Y.H., L.H.H., S.D., D.X.B.-G., Y.Z., M.F., J.D., C.M., C.A., C.G.B.), Neurogenetics Branch, NINDS, NIH, Bethesda, MD; Division of Neurology (M.E.L.), Oregon Health and Science University, Portland, OR; Department of Neurology (L.H.H.), Boston Children's Hospital, MA; Rehabilitation Medicine Department (M.S.J., M.W.); Occupational Therapy Section (R.P.), Rehabilitation Medicine Department, NIH, Bethesda, MD; Division of Neurology (D.X.B.-G.), Children's National Hospital, Washington, DC; Division of Pulmonology (O.M.), Children's Hospital of Philadelphia, PA; Santhera Pharmaceuticals (R.H., D.P.), Pratteln, Switzerland; and Department of Biostatistics (K.C.), Mailman School of Public Health, Columbia University, NY., Petraki D; From the Neuromuscular and Neurogenetics Disorders of Childhood Section (A.R.F., P.Y., M.E.L., S.B.N., G.V.A., Y.H., L.H.H., S.D., D.X.B.-G., Y.Z., M.F., J.D., C.M., C.A., C.G.B.), Neurogenetics Branch, NINDS, NIH, Bethesda, MD; Division of Neurology (M.E.L.), Oregon Health and Science University, Portland, OR; Department of Neurology (L.H.H.), Boston Children's Hospital, MA; Rehabilitation Medicine Department (M.S.J., M.W.); Occupational Therapy Section (R.P.), Rehabilitation Medicine Department, NIH, Bethesda, MD; Division of Neurology (D.X.B.-G.), Children's National Hospital, Washington, DC; Division of Pulmonology (O.M.), Children's Hospital of Philadelphia, PA; Santhera Pharmaceuticals (R.H., D.P.), Pratteln, Switzerland; and Department of Biostatistics (K.C.), Mailman School of Public Health, Columbia University, NY., Cheung K; From the Neuromuscular and Neurogenetics Disorders of Childhood Section (A.R.F., P.Y., M.E.L., S.B.N., G.V.A., Y.H., L.H.H., S.D., D.X.B.-G., Y.Z., M.F., J.D., C.M., C.A., C.G.B.), Neurogenetics Branch, NINDS, NIH, Bethesda, MD; Division of Neurology (M.E.L.), Oregon Health and Science University, Portland, OR; Department of Neurology (L.H.H.), Boston Children's Hospital, MA; Rehabilitation Medicine Department (M.S.J., M.W.); Occupational Therapy Section (R.P.), Rehabilitation Medicine Department, NIH, Bethesda, MD; Division of Neurology (D.X.B.-G.), Children's National Hospital, Washington, DC; Division of Pulmonology (O.M.), Children's Hospital of Philadelphia, PA; Santhera Pharmaceuticals (R.H., D.P.), Pratteln, Switzerland; and Department of Biostatistics (K.C.), Mailman School of Public Health, Columbia University, NY., Bönnemann CG; From the Neuromuscular and Neurogenetics Disorders of Childhood Section (A.R.F., P.Y., M.E.L., S.B.N., G.V.A., Y.H., L.H.H., S.D., D.X.B.-G., Y.Z., M.F., J.D., C.M., C.A., C.G.B.), Neurogenetics Branch, NINDS, NIH, Bethesda, MD; Division of Neurology (M.E.L.), Oregon Health and Science University, Portland, OR; Department of Neurology (L.H.H.), Boston Children's Hospital, MA; Rehabilitation Medicine Department (M.S.J., M.W.); Occupational Therapy Section (R.P.), Rehabilitation Medicine Department, NIH, Bethesda, MD; Division of Neurology (D.X.B.-G.), Children's National Hospital, Washington, DC; Division of Pulmonology (O.M.), Children's Hospital of Philadelphia, PA; Santhera Pharmaceuticals (R.H., D.P.), Pratteln, Switzerland; and Department of Biostatistics (K.C.), Mailman School of Public Health, Columbia University, NY.
Publikováno v:
Neurology. Genetics [Neurol Genet] 2024 May 29; Vol. 10 (3), pp. e200148. Date of Electronic Publication: 2024 May 29 (Print Publication: 2024).
Autor:
Bharucha-Goebel DX; From the Neuromuscular and Neurogenetic Disorders of Childhood Section (D.X.B.-G., J.J.T., D.S., A.R.F., P.M., C.G.B), National Institute of Neurological Disorders and Stroke (G.N., T.L., J.D.H.), the Rehabilitation Medicine Department, Clinical Center (M.J., M.W.), National Eye Institute (W.M.Z., L.A.H.), and the National Institute of Allergy and Infectious Diseases, Division of Intramural Research (E.M.K.), National Institutes of Health, Bethesda, and the Departments of Neurology (C.J.S., A.H., T.O.C.), Neuroscience (C.J.S., A.H.), and Pediatrics (T.O.C.), Johns Hopkins University School of Medicine, Baltimore - all in Maryland; Children's National Hospital, Washington, DC (D.X.B.-G.); the University of Iowa, Iowa City (D.S.); the Department of Pediatrics and Center for Alzheimer's and Neurodegenerative Diseases, University of Texas Southwestern Medical Center (R.M.B, S.J.G.), and Taysha Gene Therapies (E.T.) - both in Dallas; the Gene Therapy Program, University of Pennsylvania Perelman School of Medicine, Philadelphia (J.A.C.), Cencora PharmaLex, Conshohocken (B.P.C.), and Atorus Research, Newtown Square (B.S.) - all in Pennsylvania; Affinia Therapeutics, Waltham (R.C.), and the Rare Disease Research Unit, Pfizer, Cambridge (L.C., D.R.) - both in Massachusetts; the Departments of Pathology and Laboratory Medicine (D.A., T.W.B.) and Radiology (D.A.), University of North Carolina at Chapel Hill School of Medicine, Chapel Hill; and the Department of Pathology, Stanford University School of Medicine, Stanford, CA (J.E.H.)., Todd JJ; From the Neuromuscular and Neurogenetic Disorders of Childhood Section (D.X.B.-G., J.J.T., D.S., A.R.F., P.M., C.G.B), National Institute of Neurological Disorders and Stroke (G.N., T.L., J.D.H.), the Rehabilitation Medicine Department, Clinical Center (M.J., M.W.), National Eye Institute (W.M.Z., L.A.H.), and the National Institute of Allergy and Infectious Diseases, Division of Intramural Research (E.M.K.), National Institutes of Health, Bethesda, and the Departments of Neurology (C.J.S., A.H., T.O.C.), Neuroscience (C.J.S., A.H.), and Pediatrics (T.O.C.), Johns Hopkins University School of Medicine, Baltimore - all in Maryland; Children's National Hospital, Washington, DC (D.X.B.-G.); the University of Iowa, Iowa City (D.S.); the Department of Pediatrics and Center for Alzheimer's and Neurodegenerative Diseases, University of Texas Southwestern Medical Center (R.M.B, S.J.G.), and Taysha Gene Therapies (E.T.) - both in Dallas; the Gene Therapy Program, University of Pennsylvania Perelman School of Medicine, Philadelphia (J.A.C.), Cencora PharmaLex, Conshohocken (B.P.C.), and Atorus Research, Newtown Square (B.S.) - all in Pennsylvania; Affinia Therapeutics, Waltham (R.C.), and the Rare Disease Research Unit, Pfizer, Cambridge (L.C., D.R.) - both in Massachusetts; the Departments of Pathology and Laboratory Medicine (D.A., T.W.B.) and Radiology (D.A.), University of North Carolina at Chapel Hill School of Medicine, Chapel Hill; and the Department of Pathology, Stanford University School of Medicine, Stanford, CA (J.E.H.)., Saade D; From the Neuromuscular and Neurogenetic Disorders of Childhood Section (D.X.B.-G., J.J.T., D.S., A.R.F., P.M., C.G.B), National Institute of Neurological Disorders and Stroke (G.N., T.L., J.D.H.), the Rehabilitation Medicine Department, Clinical Center (M.J., M.W.), National Eye Institute (W.M.Z., L.A.H.), and the National Institute of Allergy and Infectious Diseases, Division of Intramural Research (E.M.K.), National Institutes of Health, Bethesda, and the Departments of Neurology (C.J.S., A.H., T.O.C.), Neuroscience (C.J.S., A.H.), and Pediatrics (T.O.C.), Johns Hopkins University School of Medicine, Baltimore - all in Maryland; Children's National Hospital, Washington, DC (D.X.B.-G.); the University of Iowa, Iowa City (D.S.); the Department of Pediatrics and Center for Alzheimer's and Neurodegenerative Diseases, University of Texas Southwestern Medical Center (R.M.B, S.J.G.), and Taysha Gene Therapies (E.T.) - both in Dallas; the Gene Therapy Program, University of Pennsylvania Perelman School of Medicine, Philadelphia (J.A.C.), Cencora PharmaLex, Conshohocken (B.P.C.), and Atorus Research, Newtown Square (B.S.) - all in Pennsylvania; Affinia Therapeutics, Waltham (R.C.), and the Rare Disease Research Unit, Pfizer, Cambridge (L.C., D.R.) - both in Massachusetts; the Departments of Pathology and Laboratory Medicine (D.A., T.W.B.) and Radiology (D.A.), University of North Carolina at Chapel Hill School of Medicine, Chapel Hill; and the Department of Pathology, Stanford University School of Medicine, Stanford, CA (J.E.H.)., Norato G; From the Neuromuscular and Neurogenetic Disorders of Childhood Section (D.X.B.-G., J.J.T., D.S., A.R.F., P.M., C.G.B), National Institute of Neurological Disorders and Stroke (G.N., T.L., J.D.H.), the Rehabilitation Medicine Department, Clinical Center (M.J., M.W.), National Eye Institute (W.M.Z., L.A.H.), and the National Institute of Allergy and Infectious Diseases, Division of Intramural Research (E.M.K.), National Institutes of Health, Bethesda, and the Departments of Neurology (C.J.S., A.H., T.O.C.), Neuroscience (C.J.S., A.H.), and Pediatrics (T.O.C.), Johns Hopkins University School of Medicine, Baltimore - all in Maryland; Children's National Hospital, Washington, DC (D.X.B.-G.); the University of Iowa, Iowa City (D.S.); the Department of Pediatrics and Center for Alzheimer's and Neurodegenerative Diseases, University of Texas Southwestern Medical Center (R.M.B, S.J.G.), and Taysha Gene Therapies (E.T.) - both in Dallas; the Gene Therapy Program, University of Pennsylvania Perelman School of Medicine, Philadelphia (J.A.C.), Cencora PharmaLex, Conshohocken (B.P.C.), and Atorus Research, Newtown Square (B.S.) - all in Pennsylvania; Affinia Therapeutics, Waltham (R.C.), and the Rare Disease Research Unit, Pfizer, Cambridge (L.C., D.R.) - both in Massachusetts; the Departments of Pathology and Laboratory Medicine (D.A., T.W.B.) and Radiology (D.A.), University of North Carolina at Chapel Hill School of Medicine, Chapel Hill; and the Department of Pathology, Stanford University School of Medicine, Stanford, CA (J.E.H.)., Jain M; From the Neuromuscular and Neurogenetic Disorders of Childhood Section (D.X.B.-G., J.J.T., D.S., A.R.F., P.M., C.G.B), National Institute of Neurological Disorders and Stroke (G.N., T.L., J.D.H.), the Rehabilitation Medicine Department, Clinical Center (M.J., M.W.), National Eye Institute (W.M.Z., L.A.H.), and the National Institute of Allergy and Infectious Diseases, Division of Intramural Research (E.M.K.), National Institutes of Health, Bethesda, and the Departments of Neurology (C.J.S., A.H., T.O.C.), Neuroscience (C.J.S., A.H.), and Pediatrics (T.O.C.), Johns Hopkins University School of Medicine, Baltimore - all in Maryland; Children's National Hospital, Washington, DC (D.X.B.-G.); the University of Iowa, Iowa City (D.S.); the Department of Pediatrics and Center for Alzheimer's and Neurodegenerative Diseases, University of Texas Southwestern Medical Center (R.M.B, S.J.G.), and Taysha Gene Therapies (E.T.) - both in Dallas; the Gene Therapy Program, University of Pennsylvania Perelman School of Medicine, Philadelphia (J.A.C.), Cencora PharmaLex, Conshohocken (B.P.C.), and Atorus Research, Newtown Square (B.S.) - all in Pennsylvania; Affinia Therapeutics, Waltham (R.C.), and the Rare Disease Research Unit, Pfizer, Cambridge (L.C., D.R.) - both in Massachusetts; the Departments of Pathology and Laboratory Medicine (D.A., T.W.B.) and Radiology (D.A.), University of North Carolina at Chapel Hill School of Medicine, Chapel Hill; and the Department of Pathology, Stanford University School of Medicine, Stanford, CA (J.E.H.)., Lehky T; From the Neuromuscular and Neurogenetic Disorders of Childhood Section (D.X.B.-G., J.J.T., D.S., A.R.F., P.M., C.G.B), National Institute of Neurological Disorders and Stroke (G.N., T.L., J.D.H.), the Rehabilitation Medicine Department, Clinical Center (M.J., M.W.), National Eye Institute (W.M.Z., L.A.H.), and the National Institute of Allergy and Infectious Diseases, Division of Intramural Research (E.M.K.), National Institutes of Health, Bethesda, and the Departments of Neurology (C.J.S., A.H., T.O.C.), Neuroscience (C.J.S., A.H.), and Pediatrics (T.O.C.), Johns Hopkins University School of Medicine, Baltimore - all in Maryland; Children's National Hospital, Washington, DC (D.X.B.-G.); the University of Iowa, Iowa City (D.S.); the Department of Pediatrics and Center for Alzheimer's and Neurodegenerative Diseases, University of Texas Southwestern Medical Center (R.M.B, S.J.G.), and Taysha Gene Therapies (E.T.) - both in Dallas; the Gene Therapy Program, University of Pennsylvania Perelman School of Medicine, Philadelphia (J.A.C.), Cencora PharmaLex, Conshohocken (B.P.C.), and Atorus Research, Newtown Square (B.S.) - all in Pennsylvania; Affinia Therapeutics, Waltham (R.C.), and the Rare Disease Research Unit, Pfizer, Cambridge (L.C., D.R.) - both in Massachusetts; the Departments of Pathology and Laboratory Medicine (D.A., T.W.B.) and Radiology (D.A.), University of North Carolina at Chapel Hill School of Medicine, Chapel Hill; and the Department of Pathology, Stanford University School of Medicine, Stanford, CA (J.E.H.)., Bailey RM; From the Neuromuscular and Neurogenetic Disorders of Childhood Section (D.X.B.-G., J.J.T., D.S., A.R.F., P.M., C.G.B), National Institute of Neurological Disorders and Stroke (G.N., T.L., J.D.H.), the Rehabilitation Medicine Department, Clinical Center (M.J., M.W.), National Eye Institute (W.M.Z., L.A.H.), and the National Institute of Allergy and Infectious Diseases, Division of Intramural Research (E.M.K.), National Institutes of Health, Bethesda, and the Departments of Neurology (C.J.S., A.H., T.O.C.), Neuroscience (C.J.S., A.H.), and Pediatrics (T.O.C.), Johns Hopkins University School of Medicine, Baltimore - all in Maryland; Children's National Hospital, Washington, DC (D.X.B.-G.); the University of Iowa, Iowa City (D.S.); the Department of Pediatrics and Center for Alzheimer's and Neurodegenerative Diseases, University of Texas Southwestern Medical Center (R.M.B, S.J.G.), and Taysha Gene Therapies (E.T.) - both in Dallas; the Gene Therapy Program, University of Pennsylvania Perelman School of Medicine, Philadelphia (J.A.C.), Cencora PharmaLex, Conshohocken (B.P.C.), and Atorus Research, Newtown Square (B.S.) - all in Pennsylvania; Affinia Therapeutics, Waltham (R.C.), and the Rare Disease Research Unit, Pfizer, Cambridge (L.C., D.R.) - both in Massachusetts; the Departments of Pathology and Laboratory Medicine (D.A., T.W.B.) and Radiology (D.A.), University of North Carolina at Chapel Hill School of Medicine, Chapel Hill; and the Department of Pathology, Stanford University School of Medicine, Stanford, CA (J.E.H.)., Chichester JA; From the Neuromuscular and Neurogenetic Disorders of Childhood Section (D.X.B.-G., J.J.T., D.S., A.R.F., P.M., C.G.B), National Institute of Neurological Disorders and Stroke (G.N., T.L., J.D.H.), the Rehabilitation Medicine Department, Clinical Center (M.J., M.W.), National Eye Institute (W.M.Z., L.A.H.), and the National Institute of Allergy and Infectious Diseases, Division of Intramural Research (E.M.K.), National Institutes of Health, Bethesda, and the Departments of Neurology (C.J.S., A.H., T.O.C.), Neuroscience (C.J.S., A.H.), and Pediatrics (T.O.C.), Johns Hopkins University School of Medicine, Baltimore - all in Maryland; Children's National Hospital, Washington, DC (D.X.B.-G.); the University of Iowa, Iowa City (D.S.); the Department of Pediatrics and Center for Alzheimer's and Neurodegenerative Diseases, University of Texas Southwestern Medical Center (R.M.B, S.J.G.), and Taysha Gene Therapies (E.T.) - both in Dallas; the Gene Therapy Program, University of Pennsylvania Perelman School of Medicine, Philadelphia (J.A.C.), Cencora PharmaLex, Conshohocken (B.P.C.), and Atorus Research, Newtown Square (B.S.) - all in Pennsylvania; Affinia Therapeutics, Waltham (R.C.), and the Rare Disease Research Unit, Pfizer, Cambridge (L.C., D.R.) - both in Massachusetts; the Departments of Pathology and Laboratory Medicine (D.A., T.W.B.) and Radiology (D.A.), University of North Carolina at Chapel Hill School of Medicine, Chapel Hill; and the Department of Pathology, Stanford University School of Medicine, Stanford, CA (J.E.H.)., Calcedo R; From the Neuromuscular and Neurogenetic Disorders of Childhood Section (D.X.B.-G., J.J.T., D.S., A.R.F., P.M., C.G.B), National Institute of Neurological Disorders and Stroke (G.N., T.L., J.D.H.), the Rehabilitation Medicine Department, Clinical Center (M.J., M.W.), National Eye Institute (W.M.Z., L.A.H.), and the National Institute of Allergy and Infectious Diseases, Division of Intramural Research (E.M.K.), National Institutes of Health, Bethesda, and the Departments of Neurology (C.J.S., A.H., T.O.C.), Neuroscience (C.J.S., A.H.), and Pediatrics (T.O.C.), Johns Hopkins University School of Medicine, Baltimore - all in Maryland; Children's National Hospital, Washington, DC (D.X.B.-G.); the University of Iowa, Iowa City (D.S.); the Department of Pediatrics and Center for Alzheimer's and Neurodegenerative Diseases, University of Texas Southwestern Medical Center (R.M.B, S.J.G.), and Taysha Gene Therapies (E.T.) - both in Dallas; the Gene Therapy Program, University of Pennsylvania Perelman School of Medicine, Philadelphia (J.A.C.), Cencora PharmaLex, Conshohocken (B.P.C.), and Atorus Research, Newtown Square (B.S.) - all in Pennsylvania; Affinia Therapeutics, Waltham (R.C.), and the Rare Disease Research Unit, Pfizer, Cambridge (L.C., D.R.) - both in Massachusetts; the Departments of Pathology and Laboratory Medicine (D.A., T.W.B.) and Radiology (D.A.), University of North Carolina at Chapel Hill School of Medicine, Chapel Hill; and the Department of Pathology, Stanford University School of Medicine, Stanford, CA (J.E.H.)., Armao D; From the Neuromuscular and Neurogenetic Disorders of Childhood Section (D.X.B.-G., J.J.T., D.S., A.R.F., P.M., C.G.B), National Institute of Neurological Disorders and Stroke (G.N., T.L., J.D.H.), the Rehabilitation Medicine Department, Clinical Center (M.J., M.W.), National Eye Institute (W.M.Z., L.A.H.), and the National Institute of Allergy and Infectious Diseases, Division of Intramural Research (E.M.K.), National Institutes of Health, Bethesda, and the Departments of Neurology (C.J.S., A.H., T.O.C.), Neuroscience (C.J.S., A.H.), and Pediatrics (T.O.C.), Johns Hopkins University School of Medicine, Baltimore - all in Maryland; Children's National Hospital, Washington, DC (D.X.B.-G.); the University of Iowa, Iowa City (D.S.); the Department of Pediatrics and Center for Alzheimer's and Neurodegenerative Diseases, University of Texas Southwestern Medical Center (R.M.B, S.J.G.), and Taysha Gene Therapies (E.T.) - both in Dallas; the Gene Therapy Program, University of Pennsylvania Perelman School of Medicine, Philadelphia (J.A.C.), Cencora PharmaLex, Conshohocken (B.P.C.), and Atorus Research, Newtown Square (B.S.) - all in Pennsylvania; Affinia Therapeutics, Waltham (R.C.), and the Rare Disease Research Unit, Pfizer, Cambridge (L.C., D.R.) - both in Massachusetts; the Departments of Pathology and Laboratory Medicine (D.A., T.W.B.) and Radiology (D.A.), University of North Carolina at Chapel Hill School of Medicine, Chapel Hill; and the Department of Pathology, Stanford University School of Medicine, Stanford, CA (J.E.H.)., Foley AR; From the Neuromuscular and Neurogenetic Disorders of Childhood Section (D.X.B.-G., J.J.T., D.S., A.R.F., P.M., C.G.B), National Institute of Neurological Disorders and Stroke (G.N., T.L., J.D.H.), the Rehabilitation Medicine Department, Clinical Center (M.J., M.W.), National Eye Institute (W.M.Z., L.A.H.), and the National Institute of Allergy and Infectious Diseases, Division of Intramural Research (E.M.K.), National Institutes of Health, Bethesda, and the Departments of Neurology (C.J.S., A.H., T.O.C.), Neuroscience (C.J.S., A.H.), and Pediatrics (T.O.C.), Johns Hopkins University School of Medicine, Baltimore - all in Maryland; Children's National Hospital, Washington, DC (D.X.B.-G.); the University of Iowa, Iowa City (D.S.); the Department of Pediatrics and Center for Alzheimer's and Neurodegenerative Diseases, University of Texas Southwestern Medical Center (R.M.B, S.J.G.), and Taysha Gene Therapies (E.T.) - both in Dallas; the Gene Therapy Program, University of Pennsylvania Perelman School of Medicine, Philadelphia (J.A.C.), Cencora PharmaLex, Conshohocken (B.P.C.), and Atorus Research, Newtown Square (B.S.) - all in Pennsylvania; Affinia Therapeutics, Waltham (R.C.), and the Rare Disease Research Unit, Pfizer, Cambridge (L.C., D.R.) - both in Massachusetts; the Departments of Pathology and Laboratory Medicine (D.A., T.W.B.) and Radiology (D.A.), University of North Carolina at Chapel Hill School of Medicine, Chapel Hill; and the Department of Pathology, Stanford University School of Medicine, Stanford, CA (J.E.H.)., Mohassel P; From the Neuromuscular and Neurogenetic Disorders of Childhood Section (D.X.B.-G., J.J.T., D.S., A.R.F., P.M., C.G.B), National Institute of Neurological Disorders and Stroke (G.N., T.L., J.D.H.), the Rehabilitation Medicine Department, Clinical Center (M.J., M.W.), National Eye Institute (W.M.Z., L.A.H.), and the National Institute of Allergy and Infectious Diseases, Division of Intramural Research (E.M.K.), National Institutes of Health, Bethesda, and the Departments of Neurology (C.J.S., A.H., T.O.C.), Neuroscience (C.J.S., A.H.), and Pediatrics (T.O.C.), Johns Hopkins University School of Medicine, Baltimore - all in Maryland; Children's National Hospital, Washington, DC (D.X.B.-G.); the University of Iowa, Iowa City (D.S.); the Department of Pediatrics and Center for Alzheimer's and Neurodegenerative Diseases, University of Texas Southwestern Medical Center (R.M.B, S.J.G.), and Taysha Gene Therapies (E.T.) - both in Dallas; the Gene Therapy Program, University of Pennsylvania Perelman School of Medicine, Philadelphia (J.A.C.), Cencora PharmaLex, Conshohocken (B.P.C.), and Atorus Research, Newtown Square (B.S.) - all in Pennsylvania; Affinia Therapeutics, Waltham (R.C.), and the Rare Disease Research Unit, Pfizer, Cambridge (L.C., D.R.) - both in Massachusetts; the Departments of Pathology and Laboratory Medicine (D.A., T.W.B.) and Radiology (D.A.), University of North Carolina at Chapel Hill School of Medicine, Chapel Hill; and the Department of Pathology, Stanford University School of Medicine, Stanford, CA (J.E.H.)., Tesfaye E; From the Neuromuscular and Neurogenetic Disorders of Childhood Section (D.X.B.-G., J.J.T., D.S., A.R.F., P.M., C.G.B), National Institute of Neurological Disorders and Stroke (G.N., T.L., J.D.H.), the Rehabilitation Medicine Department, Clinical Center (M.J., M.W.), National Eye Institute (W.M.Z., L.A.H.), and the National Institute of Allergy and Infectious Diseases, Division of Intramural Research (E.M.K.), National Institutes of Health, Bethesda, and the Departments of Neurology (C.J.S., A.H., T.O.C.), Neuroscience (C.J.S., A.H.), and Pediatrics (T.O.C.), Johns Hopkins University School of Medicine, Baltimore - all in Maryland; Children's National Hospital, Washington, DC (D.X.B.-G.); the University of Iowa, Iowa City (D.S.); the Department of Pediatrics and Center for Alzheimer's and Neurodegenerative Diseases, University of Texas Southwestern Medical Center (R.M.B, S.J.G.), and Taysha Gene Therapies (E.T.) - both in Dallas; the Gene Therapy Program, University of Pennsylvania Perelman School of Medicine, Philadelphia (J.A.C.), Cencora PharmaLex, Conshohocken (B.P.C.), and Atorus Research, Newtown Square (B.S.) - all in Pennsylvania; Affinia Therapeutics, Waltham (R.C.), and the Rare Disease Research Unit, Pfizer, Cambridge (L.C., D.R.) - both in Massachusetts; the Departments of Pathology and Laboratory Medicine (D.A., T.W.B.) and Radiology (D.A.), University of North Carolina at Chapel Hill School of Medicine, Chapel Hill; and the Department of Pathology, Stanford University School of Medicine, Stanford, CA (J.E.H.)., Carlin BP; From the Neuromuscular and Neurogenetic Disorders of Childhood Section (D.X.B.-G., J.J.T., D.S., A.R.F., P.M., C.G.B), National Institute of Neurological Disorders and Stroke (G.N., T.L., J.D.H.), the Rehabilitation Medicine Department, Clinical Center (M.J., M.W.), National Eye Institute (W.M.Z., L.A.H.), and the National Institute of Allergy and Infectious Diseases, Division of Intramural Research (E.M.K.), National Institutes of Health, Bethesda, and the Departments of Neurology (C.J.S., A.H., T.O.C.), Neuroscience (C.J.S., A.H.), and Pediatrics (T.O.C.), Johns Hopkins University School of Medicine, Baltimore - all in Maryland; Children's National Hospital, Washington, DC (D.X.B.-G.); the University of Iowa, Iowa City (D.S.); the Department of Pediatrics and Center for Alzheimer's and Neurodegenerative Diseases, University of Texas Southwestern Medical Center (R.M.B, S.J.G.), and Taysha Gene Therapies (E.T.) - both in Dallas; the Gene Therapy Program, University of Pennsylvania Perelman School of Medicine, Philadelphia (J.A.C.), Cencora PharmaLex, Conshohocken (B.P.C.), and Atorus Research, Newtown Square (B.S.) - all in Pennsylvania; Affinia Therapeutics, Waltham (R.C.), and the Rare Disease Research Unit, Pfizer, Cambridge (L.C., D.R.) - both in Massachusetts; the Departments of Pathology and Laboratory Medicine (D.A., T.W.B.) and Radiology (D.A.), University of North Carolina at Chapel Hill School of Medicine, Chapel Hill; and the Department of Pathology, Stanford University School of Medicine, Stanford, CA (J.E.H.)., Seremula B; From the Neuromuscular and Neurogenetic Disorders of Childhood Section (D.X.B.-G., J.J.T., D.S., A.R.F., P.M., C.G.B), National Institute of Neurological Disorders and Stroke (G.N., T.L., J.D.H.), the Rehabilitation Medicine Department, Clinical Center (M.J., M.W.), National Eye Institute (W.M.Z., L.A.H.), and the National Institute of Allergy and Infectious Diseases, Division of Intramural Research (E.M.K.), National Institutes of Health, Bethesda, and the Departments of Neurology (C.J.S., A.H., T.O.C.), Neuroscience (C.J.S., A.H.), and Pediatrics (T.O.C.), Johns Hopkins University School of Medicine, Baltimore - all in Maryland; Children's National Hospital, Washington, DC (D.X.B.-G.); the University of Iowa, Iowa City (D.S.); the Department of Pediatrics and Center for Alzheimer's and Neurodegenerative Diseases, University of Texas Southwestern Medical Center (R.M.B, S.J.G.), and Taysha Gene Therapies (E.T.) - both in Dallas; the Gene Therapy Program, University of Pennsylvania Perelman School of Medicine, Philadelphia (J.A.C.), Cencora PharmaLex, Conshohocken (B.P.C.), and Atorus Research, Newtown Square (B.S.) - all in Pennsylvania; Affinia Therapeutics, Waltham (R.C.), and the Rare Disease Research Unit, Pfizer, Cambridge (L.C., D.R.) - both in Massachusetts; the Departments of Pathology and Laboratory Medicine (D.A., T.W.B.) and Radiology (D.A.), University of North Carolina at Chapel Hill School of Medicine, Chapel Hill; and the Department of Pathology, Stanford University School of Medicine, Stanford, CA (J.E.H.)., Waite M; From the Neuromuscular and Neurogenetic Disorders of Childhood Section (D.X.B.-G., J.J.T., D.S., A.R.F., P.M., C.G.B), National Institute of Neurological Disorders and Stroke (G.N., T.L., J.D.H.), the Rehabilitation Medicine Department, Clinical Center (M.J., M.W.), National Eye Institute (W.M.Z., L.A.H.), and the National Institute of Allergy and Infectious Diseases, Division of Intramural Research (E.M.K.), National Institutes of Health, Bethesda, and the Departments of Neurology (C.J.S., A.H., T.O.C.), Neuroscience (C.J.S., A.H.), and Pediatrics (T.O.C.), Johns Hopkins University School of Medicine, Baltimore - all in Maryland; Children's National Hospital, Washington, DC (D.X.B.-G.); the University of Iowa, Iowa City (D.S.); the Department of Pediatrics and Center for Alzheimer's and Neurodegenerative Diseases, University of Texas Southwestern Medical Center (R.M.B, S.J.G.), and Taysha Gene Therapies (E.T.) - both in Dallas; the Gene Therapy Program, University of Pennsylvania Perelman School of Medicine, Philadelphia (J.A.C.), Cencora PharmaLex, Conshohocken (B.P.C.), and Atorus Research, Newtown Square (B.S.) - all in Pennsylvania; Affinia Therapeutics, Waltham (R.C.), and the Rare Disease Research Unit, Pfizer, Cambridge (L.C., D.R.) - both in Massachusetts; the Departments of Pathology and Laboratory Medicine (D.A., T.W.B.) and Radiology (D.A.), University of North Carolina at Chapel Hill School of Medicine, Chapel Hill; and the Department of Pathology, Stanford University School of Medicine, Stanford, CA (J.E.H.)., Zein WM; From the Neuromuscular and Neurogenetic Disorders of Childhood Section (D.X.B.-G., J.J.T., D.S., A.R.F., P.M., C.G.B), National Institute of Neurological Disorders and Stroke (G.N., T.L., J.D.H.), the Rehabilitation Medicine Department, Clinical Center (M.J., M.W.), National Eye Institute (W.M.Z., L.A.H.), and the National Institute of Allergy and Infectious Diseases, Division of Intramural Research (E.M.K.), National Institutes of Health, Bethesda, and the Departments of Neurology (C.J.S., A.H., T.O.C.), Neuroscience (C.J.S., A.H.), and Pediatrics (T.O.C.), Johns Hopkins University School of Medicine, Baltimore - all in Maryland; Children's National Hospital, Washington, DC (D.X.B.-G.); the University of Iowa, Iowa City (D.S.); the Department of Pediatrics and Center for Alzheimer's and Neurodegenerative Diseases, University of Texas Southwestern Medical Center (R.M.B, S.J.G.), and Taysha Gene Therapies (E.T.) - both in Dallas; the Gene Therapy Program, University of Pennsylvania Perelman School of Medicine, Philadelphia (J.A.C.), Cencora PharmaLex, Conshohocken (B.P.C.), and Atorus Research, Newtown Square (B.S.) - all in Pennsylvania; Affinia Therapeutics, Waltham (R.C.), and the Rare Disease Research Unit, Pfizer, Cambridge (L.C., D.R.) - both in Massachusetts; the Departments of Pathology and Laboratory Medicine (D.A., T.W.B.) and Radiology (D.A.), University of North Carolina at Chapel Hill School of Medicine, Chapel Hill; and the Department of Pathology, Stanford University School of Medicine, Stanford, CA (J.E.H.)., Huryn LA; From the Neuromuscular and Neurogenetic Disorders of Childhood Section (D.X.B.-G., J.J.T., D.S., A.R.F., P.M., C.G.B), National Institute of Neurological Disorders and Stroke (G.N., T.L., J.D.H.), the Rehabilitation Medicine Department, Clinical Center (M.J., M.W.), National Eye Institute (W.M.Z., L.A.H.), and the National Institute of Allergy and Infectious Diseases, Division of Intramural Research (E.M.K.), National Institutes of Health, Bethesda, and the Departments of Neurology (C.J.S., A.H., T.O.C.), Neuroscience (C.J.S., A.H.), and Pediatrics (T.O.C.), Johns Hopkins University School of Medicine, Baltimore - all in Maryland; Children's National Hospital, Washington, DC (D.X.B.-G.); the University of Iowa, Iowa City (D.S.); the Department of Pediatrics and Center for Alzheimer's and Neurodegenerative Diseases, University of Texas Southwestern Medical Center (R.M.B, S.J.G.), and Taysha Gene Therapies (E.T.) - both in Dallas; the Gene Therapy Program, University of Pennsylvania Perelman School of Medicine, Philadelphia (J.A.C.), Cencora PharmaLex, Conshohocken (B.P.C.), and Atorus Research, Newtown Square (B.S.) - all in Pennsylvania; Affinia Therapeutics, Waltham (R.C.), and the Rare Disease Research Unit, Pfizer, Cambridge (L.C., D.R.) - both in Massachusetts; the Departments of Pathology and Laboratory Medicine (D.A., T.W.B.) and Radiology (D.A.), University of North Carolina at Chapel Hill School of Medicine, Chapel Hill; and the Department of Pathology, Stanford University School of Medicine, Stanford, CA (J.E.H.)., Crawford TO; From the Neuromuscular and Neurogenetic Disorders of Childhood Section (D.X.B.-G., J.J.T., D.S., A.R.F., P.M., C.G.B), National Institute of Neurological Disorders and Stroke (G.N., T.L., J.D.H.), the Rehabilitation Medicine Department, Clinical Center (M.J., M.W.), National Eye Institute (W.M.Z., L.A.H.), and the National Institute of Allergy and Infectious Diseases, Division of Intramural Research (E.M.K.), National Institutes of Health, Bethesda, and the Departments of Neurology (C.J.S., A.H., T.O.C.), Neuroscience (C.J.S., A.H.), and Pediatrics (T.O.C.), Johns Hopkins University School of Medicine, Baltimore - all in Maryland; Children's National Hospital, Washington, DC (D.X.B.-G.); the University of Iowa, Iowa City (D.S.); the Department of Pediatrics and Center for Alzheimer's and Neurodegenerative Diseases, University of Texas Southwestern Medical Center (R.M.B, S.J.G.), and Taysha Gene Therapies (E.T.) - both in Dallas; the Gene Therapy Program, University of Pennsylvania Perelman School of Medicine, Philadelphia (J.A.C.), Cencora PharmaLex, Conshohocken (B.P.C.), and Atorus Research, Newtown Square (B.S.) - all in Pennsylvania; Affinia Therapeutics, Waltham (R.C.), and the Rare Disease Research Unit, Pfizer, Cambridge (L.C., D.R.) - both in Massachusetts; the Departments of Pathology and Laboratory Medicine (D.A., T.W.B.) and Radiology (D.A.), University of North Carolina at Chapel Hill School of Medicine, Chapel Hill; and the Department of Pathology, Stanford University School of Medicine, Stanford, CA (J.E.H.)., Sumner CJ; From the Neuromuscular and Neurogenetic Disorders of Childhood Section (D.X.B.-G., J.J.T., D.S., A.R.F., P.M., C.G.B), National Institute of Neurological Disorders and Stroke (G.N., T.L., J.D.H.), the Rehabilitation Medicine Department, Clinical Center (M.J., M.W.), National Eye Institute (W.M.Z., L.A.H.), and the National Institute of Allergy and Infectious Diseases, Division of Intramural Research (E.M.K.), National Institutes of Health, Bethesda, and the Departments of Neurology (C.J.S., A.H., T.O.C.), Neuroscience (C.J.S., A.H.), and Pediatrics (T.O.C.), Johns Hopkins University School of Medicine, Baltimore - all in Maryland; Children's National Hospital, Washington, DC (D.X.B.-G.); the University of Iowa, Iowa City (D.S.); the Department of Pediatrics and Center for Alzheimer's and Neurodegenerative Diseases, University of Texas Southwestern Medical Center (R.M.B, S.J.G.), and Taysha Gene Therapies (E.T.) - both in Dallas; the Gene Therapy Program, University of Pennsylvania Perelman School of Medicine, Philadelphia (J.A.C.), Cencora PharmaLex, Conshohocken (B.P.C.), and Atorus Research, Newtown Square (B.S.) - all in Pennsylvania; Affinia Therapeutics, Waltham (R.C.), and the Rare Disease Research Unit, Pfizer, Cambridge (L.C., D.R.) - both in Massachusetts; the Departments of Pathology and Laboratory Medicine (D.A., T.W.B.) and Radiology (D.A.), University of North Carolina at Chapel Hill School of Medicine, Chapel Hill; and the Department of Pathology, Stanford University School of Medicine, Stanford, CA (J.E.H.)., Hoke A; From the Neuromuscular and Neurogenetic Disorders of Childhood Section (D.X.B.-G., J.J.T., D.S., A.R.F., P.M., C.G.B), National Institute of Neurological Disorders and Stroke (G.N., T.L., J.D.H.), the Rehabilitation Medicine Department, Clinical Center (M.J., M.W.), National Eye Institute (W.M.Z., L.A.H.), and the National Institute of Allergy and Infectious Diseases, Division of Intramural Research (E.M.K.), National Institutes of Health, Bethesda, and the Departments of Neurology (C.J.S., A.H., T.O.C.), Neuroscience (C.J.S., A.H.), and Pediatrics (T.O.C.), Johns Hopkins University School of Medicine, Baltimore - all in Maryland; Children's National Hospital, Washington, DC (D.X.B.-G.); the University of Iowa, Iowa City (D.S.); the Department of Pediatrics and Center for Alzheimer's and Neurodegenerative Diseases, University of Texas Southwestern Medical Center (R.M.B, S.J.G.), and Taysha Gene Therapies (E.T.) - both in Dallas; the Gene Therapy Program, University of Pennsylvania Perelman School of Medicine, Philadelphia (J.A.C.), Cencora PharmaLex, Conshohocken (B.P.C.), and Atorus Research, Newtown Square (B.S.) - all in Pennsylvania; Affinia Therapeutics, Waltham (R.C.), and the Rare Disease Research Unit, Pfizer, Cambridge (L.C., D.R.) - both in Massachusetts; the Departments of Pathology and Laboratory Medicine (D.A., T.W.B.) and Radiology (D.A.), University of North Carolina at Chapel Hill School of Medicine, Chapel Hill; and the Department of Pathology, Stanford University School of Medicine, Stanford, CA (J.E.H.)., Heiss JD; From the Neuromuscular and Neurogenetic Disorders of Childhood Section (D.X.B.-G., J.J.T., D.S., A.R.F., P.M., C.G.B), National Institute of Neurological Disorders and Stroke (G.N., T.L., J.D.H.), the Rehabilitation Medicine Department, Clinical Center (M.J., M.W.), National Eye Institute (W.M.Z., L.A.H.), and the National Institute of Allergy and Infectious Diseases, Division of Intramural Research (E.M.K.), National Institutes of Health, Bethesda, and the Departments of Neurology (C.J.S., A.H., T.O.C.), Neuroscience (C.J.S., A.H.), and Pediatrics (T.O.C.), Johns Hopkins University School of Medicine, Baltimore - all in Maryland; Children's National Hospital, Washington, DC (D.X.B.-G.); the University of Iowa, Iowa City (D.S.); the Department of Pediatrics and Center for Alzheimer's and Neurodegenerative Diseases, University of Texas Southwestern Medical Center (R.M.B, S.J.G.), and Taysha Gene Therapies (E.T.) - both in Dallas; the Gene Therapy Program, University of Pennsylvania Perelman School of Medicine, Philadelphia (J.A.C.), Cencora PharmaLex, Conshohocken (B.P.C.), and Atorus Research, Newtown Square (B.S.) - all in Pennsylvania; Affinia Therapeutics, Waltham (R.C.), and the Rare Disease Research Unit, Pfizer, Cambridge (L.C., D.R.) - both in Massachusetts; the Departments of Pathology and Laboratory Medicine (D.A., T.W.B.) and Radiology (D.A.), University of North Carolina at Chapel Hill School of Medicine, Chapel Hill; and the Department of Pathology, Stanford University School of Medicine, Stanford, CA (J.E.H.)., Charnas L; From the Neuromuscular and Neurogenetic Disorders of Childhood Section (D.X.B.-G., J.J.T., D.S., A.R.F., P.M., C.G.B), National Institute of Neurological Disorders and Stroke (G.N., T.L., J.D.H.), the Rehabilitation Medicine Department, Clinical Center (M.J., M.W.), National Eye Institute (W.M.Z., L.A.H.), and the National Institute of Allergy and Infectious Diseases, Division of Intramural Research (E.M.K.), National Institutes of Health, Bethesda, and the Departments of Neurology (C.J.S., A.H., T.O.C.), Neuroscience (C.J.S., A.H.), and Pediatrics (T.O.C.), Johns Hopkins University School of Medicine, Baltimore - all in Maryland; Children's National Hospital, Washington, DC (D.X.B.-G.); the University of Iowa, Iowa City (D.S.); the Department of Pediatrics and Center for Alzheimer's and Neurodegenerative Diseases, University of Texas Southwestern Medical Center (R.M.B, S.J.G.), and Taysha Gene Therapies (E.T.) - both in Dallas; the Gene Therapy Program, University of Pennsylvania Perelman School of Medicine, Philadelphia (J.A.C.), Cencora PharmaLex, Conshohocken (B.P.C.), and Atorus Research, Newtown Square (B.S.) - all in Pennsylvania; Affinia Therapeutics, Waltham (R.C.), and the Rare Disease Research Unit, Pfizer, Cambridge (L.C., D.R.) - both in Massachusetts; the Departments of Pathology and Laboratory Medicine (D.A., T.W.B.) and Radiology (D.A.), University of North Carolina at Chapel Hill School of Medicine, Chapel Hill; and the Department of Pathology, Stanford University School of Medicine, Stanford, CA (J.E.H.)., Hooper JE; From the Neuromuscular and Neurogenetic Disorders of Childhood Section (D.X.B.-G., J.J.T., D.S., A.R.F., P.M., C.G.B), National Institute of Neurological Disorders and Stroke (G.N., T.L., J.D.H.), the Rehabilitation Medicine Department, Clinical Center (M.J., M.W.), National Eye Institute (W.M.Z., L.A.H.), and the National Institute of Allergy and Infectious Diseases, Division of Intramural Research (E.M.K.), National Institutes of Health, Bethesda, and the Departments of Neurology (C.J.S., A.H., T.O.C.), Neuroscience (C.J.S., A.H.), and Pediatrics (T.O.C.), Johns Hopkins University School of Medicine, Baltimore - all in Maryland; Children's National Hospital, Washington, DC (D.X.B.-G.); the University of Iowa, Iowa City (D.S.); the Department of Pediatrics and Center for Alzheimer's and Neurodegenerative Diseases, University of Texas Southwestern Medical Center (R.M.B, S.J.G.), and Taysha Gene Therapies (E.T.) - both in Dallas; the Gene Therapy Program, University of Pennsylvania Perelman School of Medicine, Philadelphia (J.A.C.), Cencora PharmaLex, Conshohocken (B.P.C.), and Atorus Research, Newtown Square (B.S.) - all in Pennsylvania; Affinia Therapeutics, Waltham (R.C.), and the Rare Disease Research Unit, Pfizer, Cambridge (L.C., D.R.) - both in Massachusetts; the Departments of Pathology and Laboratory Medicine (D.A., T.W.B.) and Radiology (D.A.), University of North Carolina at Chapel Hill School of Medicine, Chapel Hill; and the Department of Pathology, Stanford University School of Medicine, Stanford, CA (J.E.H.)., Bouldin TW; From the Neuromuscular and Neurogenetic Disorders of Childhood Section (D.X.B.-G., J.J.T., D.S., A.R.F., P.M., C.G.B), National Institute of Neurological Disorders and Stroke (G.N., T.L., J.D.H.), the Rehabilitation Medicine Department, Clinical Center (M.J., M.W.), National Eye Institute (W.M.Z., L.A.H.), and the National Institute of Allergy and Infectious Diseases, Division of Intramural Research (E.M.K.), National Institutes of Health, Bethesda, and the Departments of Neurology (C.J.S., A.H., T.O.C.), Neuroscience (C.J.S., A.H.), and Pediatrics (T.O.C.), Johns Hopkins University School of Medicine, Baltimore - all in Maryland; Children's National Hospital, Washington, DC (D.X.B.-G.); the University of Iowa, Iowa City (D.S.); the Department of Pediatrics and Center for Alzheimer's and Neurodegenerative Diseases, University of Texas Southwestern Medical Center (R.M.B, S.J.G.), and Taysha Gene Therapies (E.T.) - both in Dallas; the Gene Therapy Program, University of Pennsylvania Perelman School of Medicine, Philadelphia (J.A.C.), Cencora PharmaLex, Conshohocken (B.P.C.), and Atorus Research, Newtown Square (B.S.) - all in Pennsylvania; Affinia Therapeutics, Waltham (R.C.), and the Rare Disease Research Unit, Pfizer, Cambridge (L.C., D.R.) - both in Massachusetts; the Departments of Pathology and Laboratory Medicine (D.A., T.W.B.) and Radiology (D.A.), University of North Carolina at Chapel Hill School of Medicine, Chapel Hill; and the Department of Pathology, Stanford University School of Medicine, Stanford, CA (J.E.H.)., Kang EM; From the Neuromuscular and Neurogenetic Disorders of Childhood Section (D.X.B.-G., J.J.T., D.S., A.R.F., P.M., C.G.B), National Institute of Neurological Disorders and Stroke (G.N., T.L., J.D.H.), the Rehabilitation Medicine Department, Clinical Center (M.J., M.W.), National Eye Institute (W.M.Z., L.A.H.), and the National Institute of Allergy and Infectious Diseases, Division of Intramural Research (E.M.K.), National Institutes of Health, Bethesda, and the Departments of Neurology (C.J.S., A.H., T.O.C.), Neuroscience (C.J.S., A.H.), and Pediatrics (T.O.C.), Johns Hopkins University School of Medicine, Baltimore - all in Maryland; Children's National Hospital, Washington, DC (D.X.B.-G.); the University of Iowa, Iowa City (D.S.); the Department of Pediatrics and Center for Alzheimer's and Neurodegenerative Diseases, University of Texas Southwestern Medical Center (R.M.B, S.J.G.), and Taysha Gene Therapies (E.T.) - both in Dallas; the Gene Therapy Program, University of Pennsylvania Perelman School of Medicine, Philadelphia (J.A.C.), Cencora PharmaLex, Conshohocken (B.P.C.), and Atorus Research, Newtown Square (B.S.) - all in Pennsylvania; Affinia Therapeutics, Waltham (R.C.), and the Rare Disease Research Unit, Pfizer, Cambridge (L.C., D.R.) - both in Massachusetts; the Departments of Pathology and Laboratory Medicine (D.A., T.W.B.) and Radiology (D.A.), University of North Carolina at Chapel Hill School of Medicine, Chapel Hill; and the Department of Pathology, Stanford University School of Medicine, Stanford, CA (J.E.H.)., Rybin D; From the Neuromuscular and Neurogenetic Disorders of Childhood Section (D.X.B.-G., J.J.T., D.S., A.R.F., P.M., C.G.B), National Institute of Neurological Disorders and Stroke (G.N., T.L., J.D.H.), the Rehabilitation Medicine Department, Clinical Center (M.J., M.W.), National Eye Institute (W.M.Z., L.A.H.), and the National Institute of Allergy and Infectious Diseases, Division of Intramural Research (E.M.K.), National Institutes of Health, Bethesda, and the Departments of Neurology (C.J.S., A.H., T.O.C.), Neuroscience (C.J.S., A.H.), and Pediatrics (T.O.C.), Johns Hopkins University School of Medicine, Baltimore - all in Maryland; Children's National Hospital, Washington, DC (D.X.B.-G.); the University of Iowa, Iowa City (D.S.); the Department of Pediatrics and Center for Alzheimer's and Neurodegenerative Diseases, University of Texas Southwestern Medical Center (R.M.B, S.J.G.), and Taysha Gene Therapies (E.T.) - both in Dallas; the Gene Therapy Program, University of Pennsylvania Perelman School of Medicine, Philadelphia (J.A.C.), Cencora PharmaLex, Conshohocken (B.P.C.), and Atorus Research, Newtown Square (B.S.) - all in Pennsylvania; Affinia Therapeutics, Waltham (R.C.), and the Rare Disease Research Unit, Pfizer, Cambridge (L.C., D.R.) - both in Massachusetts; the Departments of Pathology and Laboratory Medicine (D.A., T.W.B.) and Radiology (D.A.), University of North Carolina at Chapel Hill School of Medicine, Chapel Hill; and the Department of Pathology, Stanford University School of Medicine, Stanford, CA (J.E.H.)., Gray SJ; From the Neuromuscular and Neurogenetic Disorders of Childhood Section (D.X.B.-G., J.J.T., D.S., A.R.F., P.M., C.G.B), National Institute of Neurological Disorders and Stroke (G.N., T.L., J.D.H.), the Rehabilitation Medicine Department, Clinical Center (M.J., M.W.), National Eye Institute (W.M.Z., L.A.H.), and the National Institute of Allergy and Infectious Diseases, Division of Intramural Research (E.M.K.), National Institutes of Health, Bethesda, and the Departments of Neurology (C.J.S., A.H., T.O.C.), Neuroscience (C.J.S., A.H.), and Pediatrics (T.O.C.), Johns Hopkins University School of Medicine, Baltimore - all in Maryland; Children's National Hospital, Washington, DC (D.X.B.-G.); the University of Iowa, Iowa City (D.S.); the Department of Pediatrics and Center for Alzheimer's and Neurodegenerative Diseases, University of Texas Southwestern Medical Center (R.M.B, S.J.G.), and Taysha Gene Therapies (E.T.) - both in Dallas; the Gene Therapy Program, University of Pennsylvania Perelman School of Medicine, Philadelphia (J.A.C.), Cencora PharmaLex, Conshohocken (B.P.C.), and Atorus Research, Newtown Square (B.S.) - all in Pennsylvania; Affinia Therapeutics, Waltham (R.C.), and the Rare Disease Research Unit, Pfizer, Cambridge (L.C., D.R.) - both in Massachusetts; the Departments of Pathology and Laboratory Medicine (D.A., T.W.B.) and Radiology (D.A.), University of North Carolina at Chapel Hill School of Medicine, Chapel Hill; and the Department of Pathology, Stanford University School of Medicine, Stanford, CA (J.E.H.)., Bönnemann CG; From the Neuromuscular and Neurogenetic Disorders of Childhood Section (D.X.B.-G., J.J.T., D.S., A.R.F., P.M., C.G.B), National Institute of Neurological Disorders and Stroke (G.N., T.L., J.D.H.), the Rehabilitation Medicine Department, Clinical Center (M.J., M.W.), National Eye Institute (W.M.Z., L.A.H.), and the National Institute of Allergy and Infectious Diseases, Division of Intramural Research (E.M.K.), National Institutes of Health, Bethesda, and the Departments of Neurology (C.J.S., A.H., T.O.C.), Neuroscience (C.J.S., A.H.), and Pediatrics (T.O.C.), Johns Hopkins University School of Medicine, Baltimore - all in Maryland; Children's National Hospital, Washington, DC (D.X.B.-G.); the University of Iowa, Iowa City (D.S.); the Department of Pediatrics and Center for Alzheimer's and Neurodegenerative Diseases, University of Texas Southwestern Medical Center (R.M.B, S.J.G.), and Taysha Gene Therapies (E.T.) - both in Dallas; the Gene Therapy Program, University of Pennsylvania Perelman School of Medicine, Philadelphia (J.A.C.), Cencora PharmaLex, Conshohocken (B.P.C.), and Atorus Research, Newtown Square (B.S.) - all in Pennsylvania; Affinia Therapeutics, Waltham (R.C.), and the Rare Disease Research Unit, Pfizer, Cambridge (L.C., D.R.) - both in Massachusetts; the Departments of Pathology and Laboratory Medicine (D.A., T.W.B.) and Radiology (D.A.), University of North Carolina at Chapel Hill School of Medicine, Chapel Hill; and the Department of Pathology, Stanford University School of Medicine, Stanford, CA (J.E.H.).
Publikováno v:
The New England journal of medicine [N Engl J Med] 2024 Mar 21; Vol. 390 (12), pp. 1092-1104.
Autor:
Schroth MK; Cure SMA (MKS); Clinical Care Education (JD), Cure SMA; Neurology and Pediatrics (DBG), Children's National, and National Institute of Neurological Diseases and Stroke, National Institutes of Health; Division of Neurology (WBB), Department of Pediatrics, Vanderbilt University; Department of Neurology (BTD), Boston Children's Hospital, Harvard Medical School; Department of Neurology (BHE), The Ohio State University Wexner Medical Center; Child Neurology (MVF), Indiana University; Division of Neuropediatrics (AK), Development and Rehabilitation, Department of Pediatrics, Inselspital, Bern University Hospital; Pediatric Neuromuscular (JK), Helen DeVos Children's Hospital; Neurology (CMP), Children's Hospital of the King's Daughters; Department of Pediatrics (AV), Division of Neurology, University of Arkansas for Medical Sciences, Arkansas Children's Hospital; and Department of Anesthesiology (RJG), Critical Care and Pain Medicine, Boston Children's Hospital, Harvard Medical School., Deans J; Cure SMA (MKS); Clinical Care Education (JD), Cure SMA; Neurology and Pediatrics (DBG), Children's National, and National Institute of Neurological Diseases and Stroke, National Institutes of Health; Division of Neurology (WBB), Department of Pediatrics, Vanderbilt University; Department of Neurology (BTD), Boston Children's Hospital, Harvard Medical School; Department of Neurology (BHE), The Ohio State University Wexner Medical Center; Child Neurology (MVF), Indiana University; Division of Neuropediatrics (AK), Development and Rehabilitation, Department of Pediatrics, Inselspital, Bern University Hospital; Pediatric Neuromuscular (JK), Helen DeVos Children's Hospital; Neurology (CMP), Children's Hospital of the King's Daughters; Department of Pediatrics (AV), Division of Neurology, University of Arkansas for Medical Sciences, Arkansas Children's Hospital; and Department of Anesthesiology (RJG), Critical Care and Pain Medicine, Boston Children's Hospital, Harvard Medical School., Bharucha Goebel DX; Cure SMA (MKS); Clinical Care Education (JD), Cure SMA; Neurology and Pediatrics (DBG), Children's National, and National Institute of Neurological Diseases and Stroke, National Institutes of Health; Division of Neurology (WBB), Department of Pediatrics, Vanderbilt University; Department of Neurology (BTD), Boston Children's Hospital, Harvard Medical School; Department of Neurology (BHE), The Ohio State University Wexner Medical Center; Child Neurology (MVF), Indiana University; Division of Neuropediatrics (AK), Development and Rehabilitation, Department of Pediatrics, Inselspital, Bern University Hospital; Pediatric Neuromuscular (JK), Helen DeVos Children's Hospital; Neurology (CMP), Children's Hospital of the King's Daughters; Department of Pediatrics (AV), Division of Neurology, University of Arkansas for Medical Sciences, Arkansas Children's Hospital; and Department of Anesthesiology (RJG), Critical Care and Pain Medicine, Boston Children's Hospital, Harvard Medical School., Burnette WB; Cure SMA (MKS); Clinical Care Education (JD), Cure SMA; Neurology and Pediatrics (DBG), Children's National, and National Institute of Neurological Diseases and Stroke, National Institutes of Health; Division of Neurology (WBB), Department of Pediatrics, Vanderbilt University; Department of Neurology (BTD), Boston Children's Hospital, Harvard Medical School; Department of Neurology (BHE), The Ohio State University Wexner Medical Center; Child Neurology (MVF), Indiana University; Division of Neuropediatrics (AK), Development and Rehabilitation, Department of Pediatrics, Inselspital, Bern University Hospital; Pediatric Neuromuscular (JK), Helen DeVos Children's Hospital; Neurology (CMP), Children's Hospital of the King's Daughters; Department of Pediatrics (AV), Division of Neurology, University of Arkansas for Medical Sciences, Arkansas Children's Hospital; and Department of Anesthesiology (RJG), Critical Care and Pain Medicine, Boston Children's Hospital, Harvard Medical School., Darras BT; Cure SMA (MKS); Clinical Care Education (JD), Cure SMA; Neurology and Pediatrics (DBG), Children's National, and National Institute of Neurological Diseases and Stroke, National Institutes of Health; Division of Neurology (WBB), Department of Pediatrics, Vanderbilt University; Department of Neurology (BTD), Boston Children's Hospital, Harvard Medical School; Department of Neurology (BHE), The Ohio State University Wexner Medical Center; Child Neurology (MVF), Indiana University; Division of Neuropediatrics (AK), Development and Rehabilitation, Department of Pediatrics, Inselspital, Bern University Hospital; Pediatric Neuromuscular (JK), Helen DeVos Children's Hospital; Neurology (CMP), Children's Hospital of the King's Daughters; Department of Pediatrics (AV), Division of Neurology, University of Arkansas for Medical Sciences, Arkansas Children's Hospital; and Department of Anesthesiology (RJG), Critical Care and Pain Medicine, Boston Children's Hospital, Harvard Medical School., Elsheikh BH; Cure SMA (MKS); Clinical Care Education (JD), Cure SMA; Neurology and Pediatrics (DBG), Children's National, and National Institute of Neurological Diseases and Stroke, National Institutes of Health; Division of Neurology (WBB), Department of Pediatrics, Vanderbilt University; Department of Neurology (BTD), Boston Children's Hospital, Harvard Medical School; Department of Neurology (BHE), The Ohio State University Wexner Medical Center; Child Neurology (MVF), Indiana University; Division of Neuropediatrics (AK), Development and Rehabilitation, Department of Pediatrics, Inselspital, Bern University Hospital; Pediatric Neuromuscular (JK), Helen DeVos Children's Hospital; Neurology (CMP), Children's Hospital of the King's Daughters; Department of Pediatrics (AV), Division of Neurology, University of Arkansas for Medical Sciences, Arkansas Children's Hospital; and Department of Anesthesiology (RJG), Critical Care and Pain Medicine, Boston Children's Hospital, Harvard Medical School., Felker MV; Cure SMA (MKS); Clinical Care Education (JD), Cure SMA; Neurology and Pediatrics (DBG), Children's National, and National Institute of Neurological Diseases and Stroke, National Institutes of Health; Division of Neurology (WBB), Department of Pediatrics, Vanderbilt University; Department of Neurology (BTD), Boston Children's Hospital, Harvard Medical School; Department of Neurology (BHE), The Ohio State University Wexner Medical Center; Child Neurology (MVF), Indiana University; Division of Neuropediatrics (AK), Development and Rehabilitation, Department of Pediatrics, Inselspital, Bern University Hospital; Pediatric Neuromuscular (JK), Helen DeVos Children's Hospital; Neurology (CMP), Children's Hospital of the King's Daughters; Department of Pediatrics (AV), Division of Neurology, University of Arkansas for Medical Sciences, Arkansas Children's Hospital; and Department of Anesthesiology (RJG), Critical Care and Pain Medicine, Boston Children's Hospital, Harvard Medical School., Klein A; Cure SMA (MKS); Clinical Care Education (JD), Cure SMA; Neurology and Pediatrics (DBG), Children's National, and National Institute of Neurological Diseases and Stroke, National Institutes of Health; Division of Neurology (WBB), Department of Pediatrics, Vanderbilt University; Department of Neurology (BTD), Boston Children's Hospital, Harvard Medical School; Department of Neurology (BHE), The Ohio State University Wexner Medical Center; Child Neurology (MVF), Indiana University; Division of Neuropediatrics (AK), Development and Rehabilitation, Department of Pediatrics, Inselspital, Bern University Hospital; Pediatric Neuromuscular (JK), Helen DeVos Children's Hospital; Neurology (CMP), Children's Hospital of the King's Daughters; Department of Pediatrics (AV), Division of Neurology, University of Arkansas for Medical Sciences, Arkansas Children's Hospital; and Department of Anesthesiology (RJG), Critical Care and Pain Medicine, Boston Children's Hospital, Harvard Medical School., Krueger J; Cure SMA (MKS); Clinical Care Education (JD), Cure SMA; Neurology and Pediatrics (DBG), Children's National, and National Institute of Neurological Diseases and Stroke, National Institutes of Health; Division of Neurology (WBB), Department of Pediatrics, Vanderbilt University; Department of Neurology (BTD), Boston Children's Hospital, Harvard Medical School; Department of Neurology (BHE), The Ohio State University Wexner Medical Center; Child Neurology (MVF), Indiana University; Division of Neuropediatrics (AK), Development and Rehabilitation, Department of Pediatrics, Inselspital, Bern University Hospital; Pediatric Neuromuscular (JK), Helen DeVos Children's Hospital; Neurology (CMP), Children's Hospital of the King's Daughters; Department of Pediatrics (AV), Division of Neurology, University of Arkansas for Medical Sciences, Arkansas Children's Hospital; and Department of Anesthesiology (RJG), Critical Care and Pain Medicine, Boston Children's Hospital, Harvard Medical School., Proud CM; Cure SMA (MKS); Clinical Care Education (JD), Cure SMA; Neurology and Pediatrics (DBG), Children's National, and National Institute of Neurological Diseases and Stroke, National Institutes of Health; Division of Neurology (WBB), Department of Pediatrics, Vanderbilt University; Department of Neurology (BTD), Boston Children's Hospital, Harvard Medical School; Department of Neurology (BHE), The Ohio State University Wexner Medical Center; Child Neurology (MVF), Indiana University; Division of Neuropediatrics (AK), Development and Rehabilitation, Department of Pediatrics, Inselspital, Bern University Hospital; Pediatric Neuromuscular (JK), Helen DeVos Children's Hospital; Neurology (CMP), Children's Hospital of the King's Daughters; Department of Pediatrics (AV), Division of Neurology, University of Arkansas for Medical Sciences, Arkansas Children's Hospital; and Department of Anesthesiology (RJG), Critical Care and Pain Medicine, Boston Children's Hospital, Harvard Medical School., Veerapandiyan A; Cure SMA (MKS); Clinical Care Education (JD), Cure SMA; Neurology and Pediatrics (DBG), Children's National, and National Institute of Neurological Diseases and Stroke, National Institutes of Health; Division of Neurology (WBB), Department of Pediatrics, Vanderbilt University; Department of Neurology (BTD), Boston Children's Hospital, Harvard Medical School; Department of Neurology (BHE), The Ohio State University Wexner Medical Center; Child Neurology (MVF), Indiana University; Division of Neuropediatrics (AK), Development and Rehabilitation, Department of Pediatrics, Inselspital, Bern University Hospital; Pediatric Neuromuscular (JK), Helen DeVos Children's Hospital; Neurology (CMP), Children's Hospital of the King's Daughters; Department of Pediatrics (AV), Division of Neurology, University of Arkansas for Medical Sciences, Arkansas Children's Hospital; and Department of Anesthesiology (RJG), Critical Care and Pain Medicine, Boston Children's Hospital, Harvard Medical School., Graham RJ; Cure SMA (MKS); Clinical Care Education (JD), Cure SMA; Neurology and Pediatrics (DBG), Children's National, and National Institute of Neurological Diseases and Stroke, National Institutes of Health; Division of Neurology (WBB), Department of Pediatrics, Vanderbilt University; Department of Neurology (BTD), Boston Children's Hospital, Harvard Medical School; Department of Neurology (BHE), The Ohio State University Wexner Medical Center; Child Neurology (MVF), Indiana University; Division of Neuropediatrics (AK), Development and Rehabilitation, Department of Pediatrics, Inselspital, Bern University Hospital; Pediatric Neuromuscular (JK), Helen DeVos Children's Hospital; Neurology (CMP), Children's Hospital of the King's Daughters; Department of Pediatrics (AV), Division of Neurology, University of Arkansas for Medical Sciences, Arkansas Children's Hospital; and Department of Anesthesiology (RJG), Critical Care and Pain Medicine, Boston Children's Hospital, Harvard Medical School.
Publikováno v:
Neurology. Clinical practice [Neurol Clin Pract] 2025 Feb; Vol. 15 (1), pp. e200374. Date of Electronic Publication: 2024 Oct 08.
Publikováno v:
Journal of pediatric ophthalmology and strabismus [J Pediatr Ophthalmol Strabismus] 2023 Jul-Aug; Vol. 60 (4), pp. e35-e37. Date of Electronic Publication: 2023 Jul 01.
Autor:
Llansó L; The John Walton Muscular Dystrophy Research Centre, Translational and Clinical Research Institute, Newcastle University and Newcastle Hospitals NHS Foundation Trust, Central Parkway, Newcastle Upon Tyne, UK; Hospital Clínic de Barcelona, Department of Neurology, Universitat de Barcelona, Spain., Moore U; The John Walton Muscular Dystrophy Research Centre, Translational and Clinical Research Institute, Newcastle University and Newcastle Hospitals NHS Foundation Trust, Central Parkway, Newcastle Upon Tyne, UK., Bolano-Diaz C; The John Walton Muscular Dystrophy Research Centre, Translational and Clinical Research Institute, Newcastle University and Newcastle Hospitals NHS Foundation Trust, Central Parkway, Newcastle Upon Tyne, UK., James M; The John Walton Muscular Dystrophy Research Centre, Translational and Clinical Research Institute, Newcastle University and Newcastle Hospitals NHS Foundation Trust, Central Parkway, Newcastle Upon Tyne, UK., Blamire AM; Magnetic Resonance Centre, Institute for Translational Medicine, Newcastle University., Carlier PG; AIM & CEA NMR Laboratory, Institute of Myology, Pitié-Salpêtrière University Hospital, Paris, France., Rufibach L; The Jain Foundation, Seattle, Washington, USA., Gordish-Dressman H; Center for Translational Science, Division of Biostatistics and Study Methodology, Children's National Health System, Washington, DC, USA., Boyle G; The John Walton Muscular Dystrophy Research Centre, Translational and Clinical Research Institute, Newcastle University and Newcastle Hospitals NHS Foundation Trust, Central Parkway, Newcastle Upon Tyne, UK., Hilsden H; The John Walton Muscular Dystrophy Research Centre, Translational and Clinical Research Institute, Newcastle University and Newcastle Hospitals NHS Foundation Trust, Central Parkway, Newcastle Upon Tyne, UK., Day JW; Department of Neurology and Neurological Sciences, Stanford University School of Medicine; Stanford, CA, USA., Jones KJ; Kids Neuroscience Centre, Sydney Children's Hospitals Network. Sydney Medical School, University of Sydney., Bharucha-Goebel DX; Department of Neurology Children's National Health System, Washington, DC, USA., Salort-Campana E; Neuromuscular and ALS center, La Timone Hospital, Aix-Marseille Université, Marseille, France., Pestronk A; Department of Neurology, Washington University School of Medicine, St. Louis, MO, USA., Walter MC; Friedrich-Baur-Institute, Dept. of Neurology, Ludwig-Maximilians-University of Munich, Germany., Paradas C; Neuromuscular Unit, Department of Neurology, Hospital U. Virgen del Rocío/Instituto de Biomedicina de Sevilla, Sevilla, Spain., Stojkovic T; Institut de Myologie, AP-HP, G.H. Pitié-Salpêtrière, Paris, France., Mori-Yoshimura M; Department of Neurology, National Center Hospital, National Center of Neurology and Psychiatry Tokyo, Japan., Bravver E; Carolinas Healthcare System Neurosciences Institute, Charlotte, NC, USA., Pegoraro E; Department of Neuroscience, University of Padova, Italy., Mendell JR; Nationwide Children's Hospital, Columbus, OH, USA., Straub V; The John Walton Muscular Dystrophy Research Centre, Translational and Clinical Research Institute, Newcastle University and Newcastle Hospitals NHS Foundation Trust, Central Parkway, Newcastle Upon Tyne, UK., Díaz-Manera J; The John Walton Muscular Dystrophy Research Centre, Translational and Clinical Research Institute, Newcastle University and Newcastle Hospitals NHS Foundation Trust, Central Parkway, Newcastle Upon Tyne, UK. Electronic address: Jordi.diaz-manera@newcastle.ac.uk.
Publikováno v:
Neuromuscular disorders : NMD [Neuromuscul Disord] 2023 Apr; Vol. 33 (4), pp. 349-357. Date of Electronic Publication: 2023 Mar 02.
Autor:
Moore U; The John Walton Muscular Dystrophy Research Centre, Translational and Clinical Research Institute, Newcastle University and Newcastle Hospitals NHS Foundation Trust, Newcastle upon Tyne, UK., Fernández-Simón E; The John Walton Muscular Dystrophy Research Centre, Translational and Clinical Research Institute, Newcastle University and Newcastle Hospitals NHS Foundation Trust, Newcastle upon Tyne, UK., Schiava M; The John Walton Muscular Dystrophy Research Centre, Translational and Clinical Research Institute, Newcastle University and Newcastle Hospitals NHS Foundation Trust, Newcastle upon Tyne, UK., Cox D; The John Walton Muscular Dystrophy Research Centre, Translational and Clinical Research Institute, Newcastle University and Newcastle Hospitals NHS Foundation Trust, Newcastle upon Tyne, UK., Gordish-Dressman H; Center for Translational Science, Division of Biostatistics and Study Methodology, Children's National Health System, Washington, DC, USA; Pediatrics, Epidemiology and Biostatistics, George Washington University, Washington, DC, USA., James MK; The John Walton Muscular Dystrophy Research Centre, Translational and Clinical Research Institute, Newcastle University and Newcastle Hospitals NHS Foundation Trust, Newcastle upon Tyne, UK., Mayhew A; The John Walton Muscular Dystrophy Research Centre, Translational and Clinical Research Institute, Newcastle University and Newcastle Hospitals NHS Foundation Trust, Newcastle upon Tyne, UK., Wilson I; Magnetic Resonance Centre, Translational and Clinical Research Institute, Newcastle University, Newcastle upon Tyne, UK., Guglieri M; The John Walton Muscular Dystrophy Research Centre, Translational and Clinical Research Institute, Newcastle University and Newcastle Hospitals NHS Foundation Trust, Newcastle upon Tyne, UK., Rufibach L; Jain Foundation, Seattle, WA, USA., Blamire A; Magnetic Resonance Centre, Translational and Clinical Research Institute, Newcastle University, Newcastle upon Tyne, UK., Carlier PG; University Paris-Saclay, CEA, DRF SHF, Orsay France., Mori-Yoshimura M; Department of Neurology, National Center Hospital, National Center of Neurology and Psychiatry, Tokyo, Japan., Day JW; Department of Neurology and Neurological Sciences, Stanford University School of Medicine, Stanford, CA, USA., Jones KJ; The Children's Hospital at Westmead and The University of Sydney, Sydney, NSW, Australia., Bharucha-Goebel DX; Department of Neurology, Children's National Health System, Washington, DC, USA; National Institutes of Health (NINDS), Bethesda, MD, USA., Salort-Campana E; Service des maladies neuromusculaire et de la SLA, Hôpital de La Timone, Marseille, France., Pestronk A; Department of Neurology, Washington University School of Medicine, St. Louis, MO, USA., Walter MC; Friedrich-Baur-Institute, Department of Neurology, LudwigMaximilians-University of Munich, Munich, Germany., Paradas C; Neuromuscular Unit, Department of Neurology, Hospital U. Virgen del Rocío/Instituto de Biomedicina de Sevilla, Sevilla, Spain., Stojkovic T; Centre de reference des maladies neuromusculaires, Institut de Myologie, AP-HP, Sorbonne Université, Hôpital Pitié-Salpêtrière, Paris, France., Bravver E; Neuroscience Institute, Carolinas Neuromuscular/ALS-MDA Center, Carolinas HealthCare System, Charlotte, NC, USA., Pegoraro E; Department of Neuroscience, University of Padova, Padua, Italy., Mendell JR; The Abigail Wexner Research Institute at Nationwide Children's Hospital, Columbus, OH, USA., Bushby K; The John Walton Muscular Dystrophy Research Centre, Translational and Clinical Research Institute, Newcastle University and Newcastle Hospitals NHS Foundation Trust, Newcastle upon Tyne, UK., Diaz-Manera J; The John Walton Muscular Dystrophy Research Centre, Translational and Clinical Research Institute, Newcastle University and Newcastle Hospitals NHS Foundation Trust, Newcastle upon Tyne, UK; Neuromuscular Disorders Unit, Neurology Department, Hospital de la Santa Creu I Sant Pau, Barcelona, Spain; Centro de Investigación Biomédica en Red en Enfermedades Raras (CIBERER), Madrid, Spain., Straub V; The John Walton Muscular Dystrophy Research Centre, Translational and Clinical Research Institute, Newcastle University and Newcastle Hospitals NHS Foundation Trust, Newcastle upon Tyne, UK. Electronic address: volker.straub@newcastle.ac.uk.
Publikováno v:
Neuromuscular disorders : NMD [Neuromuscul Disord] 2023 Feb; Vol. 33 (2), pp. 199-207. Date of Electronic Publication: 2023 Jan 06.
Autor:
Moore U; The John Walton Muscular Dystrophy Research Centre, Translational and Clinical Research Institute, Newcastle University and Newcastle Hospitals NHS Foundation Trust, Newcastle upon Tyne, UK., Caldas de Almeida Araújo E; NMR Laboratory, Neuromuscular Investigation Center, Institute of Myology, Paris, France.; NMR Laboratory, CEA/DRF/IBFJ/MIRCen, Paris, France., Reyngoudt H; NMR Laboratory, Neuromuscular Investigation Center, Institute of Myology, Paris, France.; NMR Laboratory, CEA/DRF/IBFJ/MIRCen, Paris, France., Gordish-Dressman H; Center for Translational Science, Division of Biostatistics and Study Methodology, Children's National Health System, Washington, DC, USA.; Pediatrics, Epidemiology and Biostatistics, George Washington University, Washington, DC, USA., Smith FE; Magnetic Resonance Centre, Translational and Clinical Research Institute, Newcastle University, Newcastle upon Tyne, UK., Wilson I; Magnetic Resonance Centre, Translational and Clinical Research Institute, Newcastle University, Newcastle upon Tyne, UK., James M; The John Walton Muscular Dystrophy Research Centre, Translational and Clinical Research Institute, Newcastle University and Newcastle Hospitals NHS Foundation Trust, Newcastle upon Tyne, UK., Mayhew A; The John Walton Muscular Dystrophy Research Centre, Translational and Clinical Research Institute, Newcastle University and Newcastle Hospitals NHS Foundation Trust, Newcastle upon Tyne, UK., Rufibach L; Jain Foundation, Seattle, WA, USA., Day JW; Department of Neurology and Neurological Sciences, Stanford University School of Medicine, Stanford, CA, USA., Jones KJ; The Children's Hospital at Westmead and The University of Sydney, Sydney, NSW, Australia., Bharucha-Goebel DX; Department of Neurology, Children's National Health System, Washington, DC, USA.; National Institutes of Health (NINDS), Bethesda, MD, USA., Salort-Campana E; Service des maladies neuromusculaire et de la SLA, Hôpital de La Timone, Marseille, France., Pestronk A; Department of Neurology, Washington University School of Medicine, St. Louis, MO, USA., Walter MC; Friedrich-Baur-Institute, Department of Neurology, Ludwig-Maximilians-University of Munich, Munich, Germany., Paradas C; Neuromuscular Unit, Department of Neurology, Hospital U. Virgen del Rocío/Instituto de Biomedicina de Sevilla, Sevilla, Spain., Stojkovic T; Centre de référence des maladies neuromusculaires, Institut de Myologie, AP-HP, Sorbonne Université, Hôpital Pitié-Salpêtrière, Paris, France., Mori-Yoshimura M; Department of Neurology, National Center Hospital, National Center of Neurology and Psychiatry, Tokyo, Japan., Bravver E; Neuroscience Institute, Carolinas Neuromuscular/ALS-MDA Center, Carolinas HealthCare System, Charlotte, NC, USA., Pegoraro E; Department of Neuroscience, University of Padova, Padua, Italy., Mendell JR; The Abigail Wexner Research Institute at Nationwide Children's Hospital, Columbus, OH, USA., Bushby K; The John Walton Muscular Dystrophy Research Centre, Translational and Clinical Research Institute, Newcastle University and Newcastle Hospitals NHS Foundation Trust, Newcastle upon Tyne, UK., Blamire AM; Magnetic Resonance Centre, Translational and Clinical Research Institute, Newcastle University, Newcastle upon Tyne, UK., Straub V; The John Walton Muscular Dystrophy Research Centre, Translational and Clinical Research Institute, Newcastle University and Newcastle Hospitals NHS Foundation Trust, Newcastle upon Tyne, UK., Carlier PG; Université Paris-Saclay, CEA, DRF, Service Hospitalier Frederic Joliot, Orsay, France., Diaz-Manera J; The John Walton Muscular Dystrophy Research Centre, Translational and Clinical Research Institute, Newcastle University and Newcastle Hospitals NHS Foundation Trust, Newcastle upon Tyne, UK.; Neuromuscular Disorders Unit, Neurology Department, Hospital de la Santa Creu i Sant Pau, Barcelona, Spain.; Centro de Investigación Biomédica en Red en Enfermedades Raras (CIBERER), Madrid, Spain.
Publikováno v:
Journal of cachexia, sarcopenia and muscle [J Cachexia Sarcopenia Muscle] 2022 Dec; Vol. 13 (6), pp. 2888-2897. Date of Electronic Publication: 2022 Sep 04.
Autor:
Reash NF; The Abigail Wexner Research Institute at Nationwide Children's Hospital, Columbus, Ohio., James MK; The John Walton Muscular Dystrophy Research Centre, Translational and Clinical Research Institute, Newcastle University and Newcastle Hospitals NHS Foundation Trust, Newcastle upon Tyne, UK., Alfano LN; The Abigail Wexner Research Institute at Nationwide Children's Hospital, Columbus, Ohio., Mayhew AG; The John Walton Muscular Dystrophy Research Centre, Translational and Clinical Research Institute, Newcastle University and Newcastle Hospitals NHS Foundation Trust, Newcastle upon Tyne, UK., Jacobs M; Center for Translational Science, Division of Biostatistics and Study Methodology, Children's National Health System, Washington, DC.; Pediatrics, Epidemiology and Biostatistics, George Washington University, Washington, DC., Iammarino MA; The Abigail Wexner Research Institute at Nationwide Children's Hospital, Columbus, Ohio., Holsten S; Neuroscience Institute, Carolinas Neuromuscular/ALS-MDA Center, Carolinas HealthCare System, Charlotte, North Carolina., Sakamoto C; Department of Physical Rehabilitation, National Center Hospital, National Center of Neurology and Psychiatry, Tokyo, Japan., Tateishi T; Department of Physical Rehabilitation, National Center Hospital, National Center of Neurology and Psychiatry, Tokyo, Japan., Yajima H; Department of Physical Rehabilitation, National Center Hospital, National Center of Neurology and Psychiatry, Tokyo, Japan., Duong T; Cooperative International Neuromuscular Research Group, Children's National Health System, Washington, DC.; Lucile Salter Packard Children's Hospital at Stanford, Neurology, Palo Alto, California.; Department of Neurology and Neurological Sciences, Stanford University School of Medicine, Stanford, California., de Wolf B; Cooperative International Neuromuscular Research Group, Children's National Health System, Washington, DC., Gee R; Lucile Salter Packard Children's Hospital at Stanford, Neurology, Palo Alto, California., Bharucha-Goebel DX; Department of Neurology Children's National Health System, Washington, DC.; National Institute of Neurological Disorders and Stroke, National Institutes of Health, Bethesda, Maryland., Bravver E; Neuroscience Institute, Carolinas Neuromuscular/ALS-MDA Center, Carolinas HealthCare System, Charlotte, North Carolina., Mori-Yoshimura M; Department of Neurology, National Center Hospital, National Center of Neurology and Psychiatry, Tokyo, Japan., Bushby K; The John Walton Muscular Dystrophy Research Centre, Translational and Clinical Research Institute, Newcastle University and Newcastle Hospitals NHS Foundation Trust, Newcastle upon Tyne, UK., Rufibach LE; The Jain Foundation, Seattle, Washington., Straub V; The John Walton Muscular Dystrophy Research Centre, Translational and Clinical Research Institute, Newcastle University and Newcastle Hospitals NHS Foundation Trust, Newcastle upon Tyne, UK., Lowes LP; The Abigail Wexner Research Institute at Nationwide Children's Hospital, Columbus, Ohio.
Publikováno v:
Muscle & nerve [Muscle Nerve] 2022 Aug; Vol. 66 (2), pp. 159-166. Date of Electronic Publication: 2022 May 20.