A majority of people have supported the urgent implementation of early screening for a genetic disease that causes severe ...

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

In the time since the Novartis gene therapy Zolgensma was approved for babies with spinal muscular atrophy, other treatments ...

Apitegromab, a fully human monoclonal antibody, works by selectively binding to the pro- and latent forms of myostatin in order to inhibit myostatin activation.

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

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 ...

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

Video: Spinal Muscular Atrophy: When is Surgical Intervention Appropriate? —How does one time surgery for scoliosis in children with pediatric spinal muscular atrophy get a maximum benefit ...

Chugai Pharma’s Evrysdi tablets 5mg receives Japanese approval to treat spinal muscular atrophy: Saturday, March 29, 2025, 11:00 Hrs [IST] Chugai Pharmaceutical Co., Ltd. announ ...

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