Rare disease and gene therapy stocks, battered in recent months by clinical and commercial setbacks, will likely benefit from the sudden departure this past week of Vinayak (Vinay) Prasad, MD, as ...

Over a whirlwind three weeks, Sarepta Therapeutics has faced tough safety questions around its commercial gene therapy ...

PTC Therapeutics faces pivotal FDA decisions for approvals that could potentially transform their long-term revenues. Click ...

But this time, the unknown is hitting the industry in a different way from when Prasad was tapped to lead CBER less than ...

A biopharmaceutical research company with ties in Andover has laid off a third of its workforce as one of the leaders in gene therapy goes through a “strategic ...

At the 2025 Alzheimer’s Association International Conference, one of the bigger splashes was made by an experimental cardiovascular drug. Newamsterdam Pharma Co. NV presented data showing that its ...

Dr. Vinay Prasad is stepping down as the Food and Drug Administration’s top vaccine regulator after a brief tenure. A ...

George Tidmarsh has only been at the Center for Drug Evaluation and Research for nine days, but will now add supervision of a ...

The last couple of weeks has been particularly distressing for Duchenne muscular dystrophy patients and their families. The ...

In the latest episode of STATus Report, Alex Hogan breaks down the confusing, heartbreaking saga of Sarepta and its gene ...

After our editorial, the agency relents to allow a Duchenne treatment.