Morgynn Buxbaum, took a spin class before developing a rare and life-threatening disease, Rhabdomyolysis. She spent one week in the hospital, and now has long-term symptoms.

Travere Therapeutics stock did suffer a bit of setback last week as the FDA moved back the anticipated decision date on a ...

The FDA recently granted orphan drug and rare pediatric disease designations to Infinant Health for a live biotherapeutic ...

Researchers say the CRISPR-based technique used could eventually be employed to treat more people with rare genetic diseases ...

Durham-based Atsena Therapeutics has received Regenerative Medicine Advanced Therapy (RMAT) designation from the U.S. Food and Drug Administration (FDA) for ATSN-201, its gene therapy product for the ...

A San Francisco boy with a rare bone marrow disease has been on the waitlist for more than a year. Gia Vang reports. A San ...

Most of the 15 million children with a rare disease have no FDA-approved treatments available to them. And when it comes to ...

A baby born with a rare and dangerous genetic disease is growing and thriving after getting an experimental gene-editing treatment made just for him. Researchers described the case in a new study, say ...

Founded in 1900, the Oswaldo Cruz Foundation has made fundamental contributions to the basic understanding of infectious disease and public health. Lise Alves reports.

Gene therapy could be the newest breakthrough for Duchenne muscular dystrophy. While it may not be a cure, it can give independence to boys diagnosed with DMD.

On a mission to expand its recently approved Tryngolza beyond the rare genetic disorder familial chylomicronemia syndrome ...

When an Omaha woman was diagnosed with a rare illness as a teenager, it affected not only her health but also her high school ...