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

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

Scholar Rock (SRRK) announced that the U.S. Food and Drug Administration FDA has accepted its Biologics License Application BLA for ...

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

Activating a protein called potassium channel Kv2.1 may help improve motor function in SMA, making it a potential SMA ...

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

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

Innovator drug price is Rs 22-72 lakh per year; Analysts forecast Natco to price generic drug at Rs 3,000 per year ...

Scholar Rock will present new Phase 3 SAPPHIRE trial data on apitegromab at the 2025 MDA conference, highlighting efficacy ...

22. 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 ...