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 ...

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

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

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

We need a different way to pay for these “miracle” drugs. One option is to have them paid for on an annual basis, spreading the cost over the patient’s estimated lifespan, with payment to stop if the ...

This is the third indication for Fabhalta after Novartis won FDA approval of the small molecule in paroxysmal nocturnal ...

Children with SMA given the gene therapy Zolgensma in infancy are maintaining motor milestones after up to 10 years, new ...

Delivery of onasemnogene abeparvovec into the intrathecal space was safe and effective for children with spinal muscular ...

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

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