Spinal muscular atrophy (SMA) is a prevalent paediatric neuromuscular disorder characterised by muscle weakness and atrophy resulting from degeneration of spinal cord anterior horn α motor neurons.
Bo Chen, Yuanlin Gong, Tengteng Zhou
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Cerebrospinal fluid characteristics of patients treated with intrathecal nusinersen for spinal muscular atrophy [PDF]
Rotem Orbach+6 more
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Aim The availability of disease-modifying therapies and newborn screening programs for spinal muscular atrophy (SMA) has generated an urgent need for reliable prognostic biomarkers to classify patients according to disease severity.
C. Panicucci+19 more
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POSB339 Real-World Adherence of Nusinersen for the Treatment of Spinal Muscular Atrophy (SMA) Using US Large Administrative Claims Dataset [PDF]
Nicole B. Johnson+4 more
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Performance fatigability in adults with spinal muscular atrophy treated long‐term with nusinersen
Persons with spinal muscular atrophy (pwSMA) report progressive muscle weakness but also reduced endurance when performing repetitive tasks in daily life, referred to as “performance fatigability” (PF).
B. Stolte+8 more
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P.166 Subcutaneous intrathecal catheter and port implants for administration of Nusinersen in patients with Spinal Muscular Atrophy [PDF]
A Bokeris, D Mcneely, J Sheriko
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Improvement of muscle strength in specific muscular regions in nusinersen-treated adult patients with 5q-spinal muscular atrophy [PDF]
Olivia Schreiber‐Katz+6 more
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An observational cohort study on pulmonary function in adult patients with 5q-spinal muscular atrophy under nusinersen therapy [PDF]
Bogdan Bjelica+4 more
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