Targeted rewriting of the epigenome promises to overcome several challenges in direct gene editing in patient therapies.

The FDA has denied that it plans to combine the Center for Drug Evaluation and Research and Center for Biologics Evaluation ...

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

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

Managing osteoporosis and bone health in Duchenne muscular dystrophy occurs in a high-risk environment for fractures due to ...

REGENXBIO Inc. (NASDAQ:RGNX) is one of the biotech stocks to buy according to Wall Street analysts. On July 10, REGENXBIO ...

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 ...

The FDA greenlit multiple new drugs this month and issued some notable label expansions, including for Eli Lilly’s Kisunla.

Indian-origin scientist Vinay Prasad resigned as the FDA's vaccine and gene therapy chief in just three months after backlash ...

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 ...