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 ...

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 ...

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

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

Sarepta Therapeutics (SRPT) stock is reeling this week following the death of a 16-year-old patient treated with its flagship ...

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

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 ...

Biogen enhances its portfolio and margins amidst market challenges and competition. Click here to find out why BIIB stock is ...

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