WVE-N531 demonstrated statistically significant improvements in muscle biopsy measures and functional measures in patients ...

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.

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

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

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

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 ...

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

For patients who feel their Botox wears off quickly, Dr. Zalka often suggests increasing the dosage slightly. “Injecting a ...

After bringing Zolgensma to market in 2019 as the first gene therapy for spinal muscular atrophy, Novartis is back with an intrathecal formulation intended for older patients.

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