FDA-approved treatments for spinal muscular atrophy differ in their mechanisms of action, safety profiles, and administration ...

The Supreme Court of India seeks responses from the Centre and Roche on making the spinal muscular atrophy treatment drug ...

Children with SMA in the U.K. can switch treatments based on practical needs and preferences without unexpected disease ...

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

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

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.

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

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

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 ...

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 ...