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

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

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

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

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 ...

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

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 ...

The bench said that public good outweighs the company’s profit as the drug is not available at an affordable price in India.

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

Posters presented at the 2025 Muscular Dystrophy Association (MDA) Clinical & Scientific Conference show that therapeutic ...