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 ...

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 ...

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 ...

A biopharmaceutical research company with ties in Andover has laid off a third of its workforce as one of the industry leaders in gene therapy goes through a “strategic restructuring.” Sarepta ...