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

BJP leader Suvendu Adhikari donated Rs 10 lakh towards the treatment of a girl from West Bengal's Nadia, who is battling ...

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

A film about a Kent boy who lives with a rare condition has been nominated for a Bafta TV award. Sky's Disability & Me ...

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

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.

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

Video: Spinal Muscular Atrophy: When is Surgical Intervention Appropriate? —How does one time surgery for scoliosis in children with pediatric spinal muscular atrophy get a maximum benefit ...

The high court noted that SMA is a debilitating disease which has no cure, and the only approved drug, Risdiplam, marketed ...

A pair of clinical data sets gives Novartis confidence in a new formulation of the company’s gene therapy Zolgensma for the ...

To Novartis, fresh late-stage data support the idea that its intrathecal drug, which has the same active ingredient as ...