PCR70 Avalglucosidase Alfa (AVA) Improves Symptoms and Functioning in Late-Onset Pompe Disease (LOPD) Patients vs Alglucosidase Alfa (ALG): Post-Hoc Analyses of Patient-Reported Outcomes (PROs) From COMET Trial

Autor: A Toscano, AT van der Ploeg, KI Berger, MM Dimachkie, B Schoser, C Gwaltney, J Msihid, A Hamed, N Thibault, L Pollissard, PS Kishnani

Publikováno v: Value in Health. 25:S403

Externí odkaz: https://explore.openaire.eu/search/publication?articleId=doi_________::610f6b89a38d23178460858f32be7ecc
https://doi.org/10.1016/j.jval.2022.09.2005

A 40-week phase 2B randomized, multicenter, double-blind, placebo-controlled study evaluating the safety and efficacy of memantine in amyotrophic lateral sclerosis.

Autor: Bhai S; University of Texas Southwestern Medical Center, Dallas, Texas, USA.; Neuromuscular Center, Institute for Exercise and Environmental Medicine, Texas Health Dallas, Dallas, Texas, USA., Levine T; Honor Health, Scottsdale, Arizona, USA., Moore D; Calico Consulting, Livermore, California, USA., Bowser R; Barrow Neurological Institute, Phoenix, Arizona, USA., Heim AJ; University of Kansas Medical Center, Kansas City, Kansas, USA., Walsh M; University of Kansas Medical Center, Kansas City, Kansas, USA., Shibani A; Nerve and Muscle Center of Texas, Houston, Texas, USA., Simmons Z; Penn State Hershey Medical Center, Hershey, Pennsylvania, USA., Grogan J; Penn State Hershey Medical Center, Hershey, Pennsylvania, USA., Goyal NA; University of California, Irvine, California, USA., Govindarajan R; University of Missouri, Columbia, Missouri, USA., Hussain Y; Austin Neuromuscular Center, Austin, Texas, USA.; University of Texas Dell Medical School, Austin, Texas, USA., Papsdorf T; Access TeleCare, Dallas, Texas, USA., Schwasinger-Schmidt T; University of Kansas School of Medicine-Wichita, Wichita, Kansas, USA., Olney N; Providence Brain and Spine Institute, Portland, Oregon, USA., Goslin K; Providence Brain and Spine Institute, Portland, Oregon, USA., Pulley M; University of Florida College of Medicine Jacksonville, Jacksonville, Florida, USA., Kasarskis E; University of Kentucky, Lexington, Kentucky, USA., Weiss M; University of Washington, Seattle, Washington, USA., Katz SW; Syneos Health, Morrisville, North Carolina, USA., Moser S; University of Missouri, Columbia, Missouri, USA., Jabari D; Cedars-Sinai Medical Center, Los Angeles, California, USA., Jawdat O; University of Kansas Medical Center, Kansas City, Kansas, USA., Statland J; University of Kansas Medical Center, Kansas City, Kansas, USA., Dimachkie MM; University of Kansas Medical Center, Kansas City, Kansas, USA., Barohn R; University of Missouri, Columbia, Missouri, USA.

Publikováno v: Muscle & nerve [Muscle Nerve] 2025 Jan; Vol. 71 (1), pp. 63-72. Date of Electronic Publication: 2024 Nov 07.

A protocol for scoping reviews on the role of whole-body and dedicated body-part magnetic resonance imaging for assessment of adult and juvenile idiopathic inflammatory myopathies.

Autor: Essouma M; Network of Immunity in Infection, Malignancy and Autoimmunity, Universal Scientific Education and Research Network, Yaounde, Cameroon., de Araujo DB; Internal Medicine Department, Universidade Federal de Pelotas, Pelotas, Rio Grande do Sul, RS, Brazil. araujodb@gmail.com.; Department of Internal Medicine, Faculdade de Medicina, Universidade Federal de Pelotas, Rua Santos Dumont, 172 / 703. 96.020-380, Pelotas, Rio Grande do Sul (RS), Brazil. araujodb@gmail.com., Day J; The Walter and Eliza Hall Institute of Medical Research, Melbourne, VIC, Australia.; Royal Melbourne Hospital, Melbourne, VIC, Australia.; The University of Melbourne, Melbourne, VIC, Australia., Conticini E; Rheumatology Unit, Department of Medicine, Surgery and Neurosciences, University of Siena, Siena, Italy., Riopel MA; Moravian College, Bethlehem, PA, USA., Elias AM; Pediatric Rheumatology Unit, Children's Institute, Faculdade de Medicina, Hospital das Clínicas HCFMUSP, Universidade de São Paulo, São Paulo, SP, Brazil., Paula VT; Radiology Department, Children's Institute, Faculdade de Medicina, Hospital das Clínicas HCFMUSP, Universidade de São Paulo, São Paulo, SP, Brazil., Omori CH; Pediatric Rheumatology Unit, Children's Institute, Faculdade de Medicina, Hospital das Clínicas HCFMUSP, Universidade de São Paulo, São Paulo, SP, Brazil., Guimarães JB; Grupo Fleury Medicina e Saúde, Universidade Federal de São Paulo, EPM-UNIFESP, São Paulo, Brazil., Gibson D; Medical Imaging, Fiona Stanley Hospital, Murdoch, Daren, Australia., Saad-Magalhaes C; Pediatric Rheumatology Unit, Department of Pathology, Botucatu Medical School, São Paulo State University, Botucatu, São Paulo, Brazil., Appenzeller S; Department of Orthopedics, Rheumatology and Traumatology, University of Campinas, Campinas, SP, Brazil., Schiffenbauer A; Environmental Autoimmunity Group, National Institute of Environmental Health Sciences, National Institutes of Health, Bethesda, MD, USA., Machado PM; Department of Neuromuscular Diseases and Centre for Rheumatology, Department of Rheumatology and Queen Square Centre for Neuromuscular Diseases, University College London, University College London Hospitals NHS Foundation Trust, London, UK., Feldman BM; Division of Rheumatology, The Hospital for Sick Children, Toronto, ON, Canada., Paik JJ; Department of Medicine, Division of Rheumatology, Johns Hopkins University School of Medicine, Baltimore, MD, USA., Christopher-Stine L; Department of Medicine, Division of Rheumatology, Johns Hopkins University School of Medicine, Baltimore, MD, USA., Rider LG; Environmental Autoimmunity Group, National Institute of Environmental Health Sciences, National Institutes of Health, Bethesda, MD, USA., Reed A; Department of Pediatrics, Division of Pediatric Rheumatology, Duke University, Durham, NC, USA., van der Kooi AJ; Department of Neurology, Amsterdam UMC, University of Amsterdam, Amsterdam Neuroscience, Amsterdam, Netherlands., Marrani E; Rheumatology Unit, ERN-ReCONNET Center, Meyer Children's Hospital IRCCS, Florence, Italy., Naddaf E; Department of Neurology, Mayo Clinic, Rochester, MN, USA., Kirkhus E; Department of Radiology, Oslo University Hospital, Oslo, Norway., Sanner H; Department of Rheumatology, Oslo University Hospital, Oslo, Norway.; Department of Health Sciences, Oslo New University College, Oslo, Norway., Bauer-Ventura I; Department of Medicine, Section of Rheumatology, University of Chicago, Chicago, IL, USA., Lilleker JB; Division of Musculoskeletal and Dermatological Sciences, Centre for Musculoskeletal Research, School of Biological Sciences, University of Manchester, Manchester, UK.; Northern Care Alliance NHS Foundation Trust, Manchester Academic Health Science Centre, Manchester, UK., Gupta L; Division of Musculoskeletal and Dermatological Sciences, Centre for Musculoskeletal Research, School of Biological Sciences, University of Manchester, Manchester, UK.; Department of Rheumatology, Royal Wolverhampton Hospitals NHS Trust, Wolverhampton, UK., Lucchini M; UOC Neurologia, Fondazione Policlinico Universitario Agostino Gemelli IRCCS, Rome, Italy.; Dipartimento di Neuroscienze, Sezione di Neurologia, Catholic University of Sacred Heart, Rome, Italy., Dimachkie MM; Department of Neurology, University of Kansas, Kansas City, KS, USA., Tolend M; Department of Diagnostic Imaging, Research Institute, The Hospital for Sick Children, University of Toronto, Toronto, ON, Canada., Arabi TMA; Pediatric Rheumatology Unit, Children's Institute, Faculdade de Medicina, Hospital das Clínicas HCFMUSP, Universidade de São Paulo, São Paulo, SP, Brazil., Moghadam-Kia S; Division of Rheumatology and Clinical Immunology, Department of Medicine, University of Pittsburgh School of Medicine, Pittsburgh, PA, USA., O'Hanlon S; The South Metropolitan Health Service of Western Australia, Murdoch, Australia., Phaneuf S; Patient representative, Putnam, USA., Shinjo SK; Division of Rheumatology, Faculdade de Medicina FMUSP, Universidade de São Paulo, São Paulo, Brazil., Doria AS; Department of Diagnostic Imaging, Research Institute, The Hospital for Sick Children, University of Toronto, Toronto, ON, Canada.

Publikováno v: Rheumatology international [Rheumatol Int] 2024 Nov; Vol. 44 (11), pp. 2403-2409. Date of Electronic Publication: 2024 Jul 08.

Resistance exercise in early-stage ALS patients, ALSFRS-R, Sickness Impact Profile ALS-19, and muscle transcriptome: a pilot study.

Autor: Jawdat O; Department of Neurology, University of Kansas Medical Center, Kansas City, USA., Rucker J; Department of Physical Therapy, Rehabilitation Science, and Athletic Training, University of Kansas Medical Center, Kansas City, USA., Nakano T; Laboratory of Neurobiology of Aging, Tokyo Metropolitan Institute for Geriatrics and Gerontology, 35-2 Sakaecho, Itabashi-ku, Tokyo, 173-0015, Japan.; Department of Applied Biological Science, Tokyo University of Agriculture and Technology, Fuchu-shi, Japan., Takeno K; Laboratory of Neurobiology of Aging, Tokyo Metropolitan Institute for Geriatrics and Gerontology, 35-2 Sakaecho, Itabashi-ku, Tokyo, 173-0015, Japan., Statland J; Department of Neurology, University of Kansas Medical Center, Kansas City, USA., Pasnoor M; Department of Neurology, University of Kansas Medical Center, Kansas City, USA., Dimachkie MM; Department of Neurology, University of Kansas Medical Center, Kansas City, USA., Sabus C; Department of Physical Therapy, Rehabilitation Science, and Athletic Training, University of Kansas Medical Center, Kansas City, USA.; Department of Rehabilitation Science, Tufts University School of Medicine, Boston, USA., Badawi Y; Department of Anatomy and Cell Biology, University of Kansas Medical Center, Kansas City, USA.; Department of Neuroscience, University of Pittsburgh, Pittsburgh, USA., Hunt SL; Department of Neurology, University of Kansas Medical Center, Kansas City, USA.; Department of Biostatistics and Data Science, University of Kansas Medical Center, Kansas City, USA., Tomioka NH; Laboratory of Neurobiology of Aging, Tokyo Metropolitan Institute for Geriatrics and Gerontology, 35-2 Sakaecho, Itabashi-ku, Tokyo, 173-0015, Japan., Gunewardena S; Department of Molecular and Integrative Physiology, University of Kansas Medical Center, Kansas City, USA., Bloomer C; Genome Sequencing Facility, University of Kansas Medical Center, Kansas City, USA., Wilkins HM; Department of Neurology, University of Kansas Medical Center, Kansas City, USA., Herbelin L; Department of Neurology, University of Kansas Medical Center, Kansas City, USA.; Department of Neurology, University of Missouri, School of Medicine, 1 Hospital Dr, Columbia, MO, 65201, USA., Barohn RJ; Department of Neurology, University of Kansas Medical Center, Kansas City, USA. rbarohn@health.missouri.edu.; Department of Neurology, University of Missouri, School of Medicine, 1 Hospital Dr, Columbia, MO, 65201, USA. rbarohn@health.missouri.edu., Nishimune H; Laboratory of Neurobiology of Aging, Tokyo Metropolitan Institute for Geriatrics and Gerontology, 35-2 Sakaecho, Itabashi-ku, Tokyo, 173-0015, Japan. HiroshiNishimune@gmail.com.; Department of Applied Biological Science, Tokyo University of Agriculture and Technology, Fuchu-shi, Japan. HiroshiNishimune@gmail.com.; Department of Anatomy and Cell Biology, University of Kansas Medical Center, Kansas City, USA. HiroshiNishimune@gmail.com.

Publikováno v: Scientific reports [Sci Rep] 2024 Sep 17; Vol. 14 (1), pp. 21729. Date of Electronic Publication: 2024 Sep 17.

Measurement properties of the Inclusion Body Myositis Functional Rating Scale.

Autor: Salam S; Department of Neuromuscular Diseases, University College London, London, UK., Symonds T; Clinical Outcomes Solutions Ltd, Folkestone, UK., Doll H; Clinical Outcomes Solutions Ltd, Folkestone, UK., Rousell S; Clinical Outcomes Solutions Ltd, Folkestone, UK., Randall J; Clinical Outcomes Solutions Ltd, Folkestone, UK., Lloyd-Price L; Clinical Outcomes Solutions Ltd, Folkestone, UK., Hudgens S; Clinical Outcomes Solutions Ltd, Tucson, Arizona, USA., Guldberg C; Orphazyme Aps, Copenhagen, Denmark., Herbelin L; Department of Neurology, University of Missouri, Columbia, Missouri, USA., Barohn RJ; Department of Neurology, University of Missouri, Columbia, Missouri, USA., Hanna MG; Department of Neuromuscular Diseases, University College London, London, UK., Dimachkie MM; Department of Neurology, University of Kansas City Medical Center, Kansas City, Missouri, USA., Machado PM; Department of Neuromuscular Diseases, University College London, London, UK p.machado@ucl.ac.uk.; NIHR University College London Hospitals Biomedical Research Centre, University College London Hospitals National Health Service (NHS) Trust, London, UK.

Publikováno v: Journal of neurology, neurosurgery, and psychiatry [J Neurol Neurosurg Psychiatry] 2024 Jul 11. Date of Electronic Publication: 2024 Jul 11.