Nusinersen treatment significantly improves hand grip strength, hand motor function and MRC sum scores in adult patients with spinal muscular atrophy types 3 and 4

Background Nusinersen recently became available as the first treatment for Spinal Muscular Atrophy (SMA) and data on its effectiveness and safety in adult SMA patients are still scarce. Methods We evaluated the effectiveness and safety of nusinersen treatment during 14 months in 16 adult patients wi...

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Bibliographic Details
Published in:Journal of neurology 2021-03, Vol.268 (3), p.923-935
Main Authors: De Wel, Bram, Goosens, Veerle, Sobota, Atka, Van Camp, Elke, Geukens, Ellen, Van Kerschaver, Griet, Jagut, Marlène, Claes, Kathleen, Claeys, Kristl G.
Format: Article
Language:English
Subjects:
Adults
Adverse events
Antisense oligonucleotides
Atrophy
Hospitals
Medical research
Medicine
Medicine & Public Health
Muscle function
Muscle strength
Natural history
Nephrology
Neurology
Neuromuscular diseases
Neuroradiology
Neurosciences
Original Communication
Patients
Proteins
Safety
Scoliosis
Spinal muscular atrophy
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Summary:Background Nusinersen recently became available as the first treatment for Spinal Muscular Atrophy (SMA) and data on its effectiveness and safety in adult SMA patients are still scarce. Methods We evaluated the effectiveness and safety of nusinersen treatment during 14 months in 16 adult patients with SMA types 3 and 4 in a prospective study, and retrospectively detailed the natural history of 48 adult SMA patients types 2, 3 and 4. Results Hand grip strength ( p  = 0.03), hand motor function ( p  = 0.04) as assessed by a sub-score of the Revised Upper Limb Module (RULM) and the Medical Research Council (MRC) sum score ( p  = 0.04) improved significantly at month 14. Importantly, the MRC sum score had declined significantly ( p  
ISSN:0340-5354
1432-1459
DOI:10.1007/s00415-020-10223-9