Keya Hatkar, a young author and motivational speaker with Spinal Muscular Atrophy, inspires others with her resilience and ...

Haris Beeran urges government intervention to make affordable SMA medicines available in India, addressing the high costs and patent issues.

Upper House member Haris Beeran has said the current costs of medicines approved for SMA are 'beyond the reach of the central ...

Novartis has reported positive topline outcomes from the Phase III STEER trial of an investigational gene therapy, ...

Novartis has recently announced that its intrathecal (IT) formulation of onasemnogene abeparvovec (OAV101 IT) successfully ...

Novartis has recently announced that its intrathecal (IT) formulation of onasemnogene abeparvovec (OAV101 IT) successfully achieved the primary endpoint in a Phase III STEER study (NCT05089656) ...

—A new study analyzed health-related quality of life (HRQoL) using 5 generic and neuromuscular disease-specific rating scales in German children with spinal muscular atrophy (SMA), most of whom ...

—Newborn screening for spinal muscular atrophy allows for earlier diagnosis in preterm infants, leading to earlier use of disease-modifying therapies, in the presymptomatic stage, and better ...

The new treatment shares the active ingredient with Novartis's gene therapy Zolgensma, which was introduced five years ago as the first potential cure for babies with spinal muscular atrophy. The new ...

Novartis AG said a new experimental medicine improved motor function in children with a form of spinal muscular atrophy, the ...