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 ...

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 ...

Learn more about whether Corcept Therapeutics Incorporated or Novartis AG is a better investment based on AAII's A+ Investor ...

Regenxbio is ranked Strong Buy due to low EV, strong partnerships, royalty income, bullish trends, and buyout potential.

Gene therapy, with its offer of a possible cure for rare diseases like sickle cell, is losing early investors to ...

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

Recall that Novartis’ (NVS) Zolgensma (approved in 2019), an AAV-based gene therapy for the treatment of spinal muscular ...

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 ...