U.S. Health Secretary Robert F. Kennedy Jr. on Tuesday added two rare genetic disorders, Duchenne Muscular Dystrophy and ...

Gene editing has the potential to cure thousands of people suffering from devastating rare diseases. But the Nobel Prize-winning technology faces a basic math problem: It’s not profitable to spend ...

Backed by a handful of prominent investors including the Gates Foundation, the startup is pursuing an approach it believes ...

The nonprofit is funding research at two universities—Oregon Health & Science University and UMass Chan Medical School—on a ...

Once seen as a last resort, bone marrow transplantation is now being viewed as a potential cure for several rare blood ...

For Americans living with rare diseases, the Rare Pediatric Disease Priority Review Voucher Program is a vital tool. Yet its reauthorization is at risk.

The FDA has granted approval to a new gene therapy for a rare immune disorder, the maker of which is notably not a drug ...

The donation from Don and Anne Edwards aims to speed research, treatment and diagnosis for children with rare and genetic ...

Ohio State student Cailin Joyce died after suffering a rare blood disorder caused by the Epstein-Barr virus that “left her ...

Backed by Italy-based Fondazione Telethon ETS, Waskyra, for Wiskott-Aldrich syndrome, is the first gene therapy from a ...

Rare growth disorders are a diverse group of uncommon conditions that affect an individual’s physical development. They may stem from genetic mutations, hormonal imbalances, or other complex factors.

PEWAUKEE, Wis. - A Pewaukee restaurant held a fundraiser on Saturday for a family whose 13-year-old son has an ...