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 ...

Niemann-Pick Disease Type C (NPC) is a rare genetic disorder caused by mutations in the NPC1 or NPC2 genes, leading to harmful lipid accumulation in organs like the liver and brain. Symptoms, often ...

Vinay Prasad’s ouster as the Food and Drug Administration’s top vaccine and gene therapy regulator came after frustration ...

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

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

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

In a striking bipartisan move, the Senate Appropriations Committee voted 26-3 to boost the NIH’s budget by $400 million — rejecting the Trump administration’s proposed $17 billion cut and sweeping ...

Jeff Yass, co-founder of ByteDance investor Susquehanna International Group, made his largest ever political donations to Trump’s MAGA Inc. super PAC in the first half of the year, per a new FEC ...