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 ...

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

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

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

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

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 ...

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.

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 ...

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 ...

Meanwhile, access to Zolgensma in the UK has been suspended in children older than 12 completed months, as there have been a "small number" of cases of liver-related side effects among children ...