Results 171 to 180 of about 7,776 (285)

The Impact of Nusinersen and Risdiplam on Motor Function for Spinal Muscular Atrophy Type 2 and 3: A Meta-Analysis.

open access: yesJournal of the College of Physicians and Surgeons--Pakistan : JCPSP
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
semanticscholar   +1 more source

Cerebrospinal fluid characteristics of patients treated with intrathecal nusinersen for spinal muscular atrophy [PDF]

open access: hybrid, 2022
Rotem Orbach   +6 more
openalex   +1 more source

Proteomics profiling and machine learning in nusinersen-treated patients with spinal muscular atrophy

open access: yesCellular and molecular life sciences : CMLS
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
semanticscholar   +1 more source

Performance fatigability in adults with spinal muscular atrophy treated long‐term with nusinersen

open access: yesMuscle and Nerve
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
semanticscholar   +1 more source

Improvement of muscle strength in specific muscular regions in nusinersen-treated adult patients with 5q-spinal muscular atrophy [PDF]

open access: gold, 2023
Olivia Schreiber‐Katz   +6 more
openalex   +1 more source

An observational cohort study on pulmonary function in adult patients with 5q-spinal muscular atrophy under nusinersen therapy [PDF]

open access: hybrid, 2023
Bogdan Bjelica   +4 more
openalex   +1 more source

Early experiences of nusinersen for the treatment of spinal muscular atrophy: Results from a large survey of patients and caregivers

open access: hybrid, 2020
Er Chen   +7 more
openalex   +1 more source

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