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 broad approval for a successor drug to its blockbuster gene therapy ...

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 ...

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 ...

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 ...

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

Gene therapy, which promises a possible cure for rare diseases like sickle cell, is losing early investors to higher-reward ...

In less than two weeks, the country came together and collected over KGS 510 million (USD 5.9 million), displaying unprecedented unity and generosity.