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 ...

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

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

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

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 ...

Allogene Therapeutics has reported a patient death tied to its anti-CD52 monoclonal antibody used for lymphodepletion in its ...