A film about a Kent boy who lives with a rare condition has been nominated for a Bafta TV award. Sky's Disability & Me ...

Intrathecal treatment with a vector-based gene therapy was associated with a greater improvement in motor function at 52 ...

The FDA has accepted for Priority Review the BLA for apitegromab for the improvement of motor function in patients with spinal muscular atrophy.

Since 2016, the FDA has approved three disease-modifying treatments for spinal muscular atrophy, with several ...

A rare genetic condition, Spinal Muscular Atrophy (SMA) affects one in 7,000 people in India. It mostly affects children and ...

BJP leader Suvendu Adhikari donated Rs 10 lakh towards the treatment of a girl from West Bengal's Nadia, who is battling ...

The high court noted that SMA is a debilitating disease which has no cure, and the only approved drug, Risdiplam, marketed ...

A Prescription Drug User Fee Act (PDUFA) target action date for apitegromab has been set for Sept. 22, 2025.

Speakers from the Muscular Dystrophy Association (MDA) highlighted 75 years of progress in understanding and treating ...

Apitegromab is an investigational treatment aimed at improving motor function for people living with spinal muscular atrophy. Scholar Rock said it has also submitted and received validation for its ...

for apitegromab for the improvement of motor function in patients with spinal muscular atrophy (SMA). Apitegromab, a fully human monoclonal antibody, works by selectively binding to the pro- and ...