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

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 ...

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

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 ...

On Wednesday, Novartis gave a more detailed look at long-awaited clinical data that the company believes will help secure a ...

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

In 2022, two patients died of acute liver failure after receiving Novartis’s gene therapy Zolgensma, which treats spinal muscular atrophy, another rare genetic disorder. Elevidys is a one-time ...

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

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 ...

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

Over 95% of babies now diagnosed with SMA are treated with Zolgensma gene therapy, per Daniel Grant, vice president for ...