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.

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 ...

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

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

Zolgensma, though, is injected into the veins, and is only cleared for use in patients under two years of age. Novartis has spent years trying to show OAV101, which is shot right into the spine ...

Novartis’ Zolgensma gene therapy for spinal muscular atrophy is the most expensive drug in the world, but that hasn’t stopped it making a strong start in the US market. Zolgensma (onasemnogene ...

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

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

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