The FDA is allowing Sarepta to resume shipments of Elevidys (delandistrogene moxeparvovec) to ambulatory patients with Duchenne muscular dystrophy.

From innovation in manufacturing to more-flexible regulation and better communication with payers, much needs to happen to ...

US Food and Drug Administration Commissioner Marty Makary said he’s trying to persuade Vinay Prasad, the agency’s former head ...

Extend transfection complex formation time by up to 3 hours, reduce complex volume by >50% and maintain high titers and full ...

Sarepta and Capricor learned of key regulatory decisions from the media and investors, and Duchenne muscular dystrophy ...

Parents of boys with Duchenne muscular dystrophy weigh in on drug innovation and medical regulation.

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 ...

The U.S. Food and Drug Administration's (FDA) top vaccine official is stepping down after just three months in a role that ...

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 ...