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Liying Cui is corresponding author. Objectives To evaluate the effectiveness of nusinersen among Chinese adults with Type II & III 5q-spinal muscular atrophy (SMA). Methods […]
Yi Wang is corresponding author. Background and objective: To assess the effectiveness and safety of nusinersen for Type II/III 5q-spinal muscular atrophy (SMA) among Chinese […]
ADSS1 myopathy is a rare, autosomal recessive disease caused by mutations in the ADSS1 gene, which encodes a striated muscle-specific isoform of adenylosuccinate synthetase (AdSS1). […]
Prediction models for natural history trajectories can provide important benchmarks for the outcomes of patients receiving novel therapies, especially over multi-year periods for which placebo […]
Sevasemten, an investigational fast skeletal myosin inhibitor designed to limit muscle damage from functional dystrophin deficits, is under investigation for treating muscular dystrophies. The Phase […]
Background: Becker muscular dystrophy (BeckerMD) is a progressive myopathy caused by pathogenic variants in the dystrophin gene. Contraction-induced injury leads to replacement of muscle by […]
Background Duchenne muscular dystrophy (DMD) is a progressive X-linked neuromuscular disorder caused by dystrophin deficiency, resulting in muscle weakness and loss of ambulation. Corticosteroids, including […]
Background Duchenne muscular dystrophy (DMD) is a progressive neuromuscular disorder caused by mutations in the dystrophin gene, leading to a complete absence of the dystrophin […]
Background: Duchenne muscular dystrophy (DMD) is a severe X-linked disorder caused by mutations in the DMD gene, leading to dystrophin deficiency and progressive muscle degeneration. […]