Every year, thousands of infants in America are diagnosed with devastating genetic diseases through newborn screenings. The ...

Good morning! Today, we get deeper into the ousting of Nicole Verdun at the FDA, see promise from Vertex’s cell therapy for ...

Capricor stock drops as FDA cancels advisory meeting on Duchenne therapy; leadership changes and regulatory tensions fuel uncertainty.

The Inflation Reduction Act includes an exemption for orphan drugs for a single indication, but experts say this is far from sufficient to maintain momentum in the rare disease space.

Panelists discuss how spinal muscular atrophy is an autosomal recessive genetic disease affecting motor neurons with 3 ...

Panelists discuss how the pooled 3-year data for delandistrogene moxeparvovec show sustained motor function stabilization and ...

Shares in both companies were down following news of the second death, casting fresh safety concerns over Elevidys.

Ahead of Kajol’s supernatural horror Maa’s premiere on June 27, here are other top films that make for an engaging watch on ...

Ian has been a Patron of Muscular Dystrophy UK since 2019 but his involvement with the charity began almost three decades ...

Nicole Verdun, MD, super office director of the FDA’s Office of Therapeutic Products (OTP) and her deputy Rachael Anatol, PhD ...