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

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

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 ...

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

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 ...

One patient with Duchenne muscular dystrophy (DMD) has passed away following treatment with Sarepta Therapeutics’ gene therapy Elevidys, the biotech ...

Novartis already markets an IV-administered version of the gene therapy called Zolgensma.The product, which is one of the most expensive drugs in the world, is used to treat SMA in children ...

Sarepta Therapeutics said early Tuesday that a patient had died after receiving its gene therapy Elevidys, which treats the rare genetic condition Duchenne muscular dystrophy. A young man with ...

Novartis added Zolgensma to its pipeline by acquiring Avexis in a deal that closed earlier this year but the problems relate to events that happened well before this when Zolgensma, originally ...

The application for Zolgensma was approved at the Central Social Insurance Medical Council, an advisory council for the health minister, on May 13 and will go into effect on May 20.