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 ...

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 ...

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 ...

In September 2020, when he was 14 months old, he and his family travelled to the Children’s Hospital of San Antonio in the US where he received his Zolgensma infusion. A year later, he was able ...

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 ...

Delivery of onasemnogene abeparvovec into the intrathecal space was safe and effective for children with spinal muscular atrophy (SMA) aged 2 to 17 years, who had previously been shut out of receiving ...

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

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