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

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 ...

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

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

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 ...

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

Sarepta Therapeutics said early Tuesday that a patient had died after receiving its gene therapy Elevidys, which treats the ...

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

Vincent Gaynor remembers, almost to the minute, when he realized his part in birthing the breakthrough gene therapy Zolgensma had ended and the forces that turned it into the world’s most ...

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 ...