Positive CHMP opinion is based on data from the DEVOTE study which evaluated the high dose regimen of nusinersen in treatment ...

Titin-based mechanosensing is a key driver of trophic signaling in muscle, yet the downstream pathways linking titin sensing to muscle remodeling remain poorly understoo ...

Lipton and his colleagues have created a new class of deployable structures that could one day be used to create artificial ...

Scholar Rock Holding Company ( SRRK) is a Boston-based, clinical-stage biotech that was founded in 2012, focusing on ...

Treatment with onasemnogene abeparvovec gene therapy for spinal muscular atrophy (SMA) type 1 was associated with less need for nutritional support or nighttime ventilation compared to recommended ...

Spinal muscular atrophy is a rare genetic disease of the nerve cells in the spinal cord that can appear as early as infancy. The disease leads to a progressive loss of muscle strength. Those affected ...

A new clinical trial has revealed encouraging results for a muscle-targeting therapy aimed at improving motor function in children and adolescents with spinal muscular atrophy, according to a study ...

Participants aged 2 to 12 years receiving apitegromab had significantly higher improvements in motor function vs placebo. HealthDay News — For patients with nonambulatory type 2 or type 3 spinal ...

Significant improvement seen in motor function for patients in combined 20- and 10-mg/kg dose groups vs placebo group. HealthDay News — For patients with nonambulatory type 2 or type 3 spinal muscular ...

A Johns Hopkins Children’s Center-led study shows that patients with spinal muscular atrophy (SMA) could regain muscle with the help of a new monoclonal antibody treatment. Researchers say the study ...

Oral risdiplam (Evrysdi, Genentech) started in the first 6 weeks of life let most infants with presymptomatic spinal muscular atrophy (SMA) reach motor milestones typical of healthy babies, results of ...

Spinal muscular atrophy (SMA) is a genetic neuromuscular disease affecting specialized nerve cells that control voluntary muscle movement, according to the Muscular Dystrophy Association (MDA). It can ...