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

Prasad, a longtime critic of the FDA’s leniency in drug approvals, joined the agency in May. WASHINGTON — The Food and Drug ...

Two major health agencies are facing leadership reshuffles, with the resignations of the FDA’s Vinay Prasad and NICE’s Dr Sam Roberts.

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

Shipment of delandistrogene moxeparvovec, a gene therapy for Duchenne muscular dystrophy (DMD), will resume following a ...

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 ...

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 ...

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