Without this protein, motor neurons deteriorate, leading to muscle weakness, impaired movement, and eventually respiratory ...

The Phase 3 STEER trial enrolled more than 100 children with SMA type 2, ages 2-17, who were able to sit, but hadn't walked ...

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

Novartis’ Zolgensma, an IV formulation, was FDA-approved in 2019 for patients under two with SMA. However, the new IT ...

A Rajya Sabha MP urges the health minister to enable local production of Risdiplam, a vital drug for spinal muscular atrophy, ...

Centre informed Supreme Court that providing free gene therapy for Spinal Muscular Atrophy (SMA) could cost India Rs ...

Scholar Rock (NASDAQ: SRRK), a late-stage biopharmaceutical company focused on advancing innovative treatments for spinal ...

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

Typically, patients do not attend these meetings, with their family or caregivers representing them, says Unnikrishnan. But, ...

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

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) ...

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