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 ...

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

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 ...

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 ...

Sarepta Therapeutics said early Tuesday that a patient had died after receiving its gene therapy Elevidys, which treats the rare genetic condition Duchenne muscular dystrophy. A young man with ...

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

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 ...

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 ...

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