Nusinersen improves walking distance and reduces fatigue in later‐onset spinal muscular atrophy
| Autor: | Allan M. Glanzman, Elena S. Mazzone, Sally Dunaway Young, Eugenio Mercuri, Wildon Farwell, Eugene Schneider, Basil T. Darras, Darryl C. De Vivo, Kathie M. Bishop, C. Frank Bennett, Richard S. Finkel, Jacqueline Montes, Francesco Muntoni, Richard Foster, Amy Pasternak |
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| Rok vydání: | 2019 |
| Předmět: |
Male
0301 basic medicine 6‐minute walk test medicine.medical_specialty Percentile Adolescent Physiology Oligonucleotides Walk Test Walking 030105 genetics & heredity Muscular Atrophy Spinal 03 medical and health sciences Cellular and Molecular Neuroscience 0302 clinical medicine Physical medicine and rehabilitation Physiology (medical) medicine Humans Child spinal muscular atrophy Aged neuromuscular junction business.industry nusinersen Infant Muscle weakness Spinal muscular atrophy medicine.disease SMA Gait Walk test Child Preschool Clinical Research Short Report Ambulatory Clinical Research Short Reports Female fatigue Nusinersen Neurology (clinical) medicine.symptom business 030217 neurology & neurosurgery |
| Zdroj: | Muscle & Nerve |
| ISSN: | 1097-4598 0148-639X |
| DOI: | 10.1002/mus.26633 |
| Popis: | Introduction Ambulatory individuals with spinal muscular atrophy (SMA) experience muscle weakness, gait impairments, and fatigue that affect their walking ability. Improvements have been observed in motor function in children treated with nusinersen, but its impact on fatigue has not been studied. Methods Post hoc analyses were used to examine changes in 6‐minute walk test (6MWT) distance and fatigue in children and adolescents with SMA type II and III who received their first dose of nusinersen in the phase Ib/IIa, open‐label CS2 study and were ambulatory during CS2 or the extension study, CS12. Results Fourteen children performed the 6MWT. Median (25th, 75th percentile) distance walked increased over time by 98.0 (62.0, 135.0) meters at day 1050, whereas median fatigue changed by −3.8% (−19.7%, 1.4%). Discussion These results support previous studies demonstrating clinically meaningful effects of nusinersen on motor function in children and adolescents with later‐onset SMA. |
| Databáze: | OpenAIRE |
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