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

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

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.

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 ...

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 ...

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 ...

—Sleep-disordered breathing occurs frequently in patients with spinal muscular atrophy and can be obstructive or non-obstructive, with those diagnosed with non-obstructive SDB having worse ...

Opens in a new tab or window How does one time surgery for scoliosis in children with pediatric spinal muscular atrophy get a maximum benefit without impeding growth? Dr. Ashley E. Webb of the ...

Novartis' OAV101 IT gene therapy showed positive results in Phase 3 trials for SMA, with data supporting its potential as a treatment option.