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

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.

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

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

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

Novartis released two Phase 3 datasets on Wednesday that show a new version of its gene therapy Zolgensma led to motor ...

Sarepta Therapeutics (SRPT) stock is reeling this week following the death of a 16-year-old patient treated with its flagship ...

Gene therapy, with its offer of a possible cure for rare diseases like sickle cell, is losing early investors to ...

The FDA wants to end bulk production of copycat versions of weight loss drugs; telehealth companies and compounding ...

Delivery of onasemnogene abeparvovec into the intrathecal space was safe and effective for children with spinal muscular ...

A young person with Duchenne muscular dystrophy died following treatment with the recently approved gene therapy ...