Amyotrophic lateral sclerosis (ALS) is a neurodegenerative disease in which premature loss of upper and lower motor neurons leads to fatal paralysis. A landmark contribution […]
Background: The Spinal Muscular Atrophy Independence Scale (SMAIS) assesses the amount of assistance required to perform daily activities for individuals with Type 2 and non-ambulant […]
Background: AMX0035 is an oral fixed-dose coformulation (sodium phenylbutyrate-taurursodiol) that significantly slowed functional decline as measured by the Amyotrophic Lateral Sclerosis Functional Rating Scale–Revised (ALSFRS-R) […]
Vamorolone is a first-in-class steroidal anti-inflammatory drug with novel structure/activity relationships with glucocorticoid and mineralocorticoid receptor targets compared to deflazacort or prednisone. Published open-label dose-finding […]
Objective: To examine physical activity and the relationships to measures of function and disease severity in individuals with CMT1A. Background: Physical activity has been reported […]
Background: Adeno-associated virus (AAV)-mediated gene transfer therapy has shown early signs of potential to treat Duchenne Muscular Dystrophy (DMD). The intent of rAAVrh74.MHCK7.micro-dystrophin (SRP-9001) is […]
Background: The American Academy of Pediatrics (AAP) recommends regular psychosocial screening for boys with Duchenne Muscular Dystrophy (DMD), stating that this population is at increased […]
Background: Apitegromab (SRK-015) is a fully human, high-affinity anti-proMyostatin monoclonal antibody that binds to human proMyostatin and latent myostatin and inhibits the tolloid-mediated proteolysis step […]
Background: Duchenne muscular dystrophy (DMD) is a fatal rare, X-linked disease characterized by progressive muscle weakness. Approximately 10–15% of DMD cases are caused by a […]