Miriya Nurse, 16, spent months in the hospital undergoing a groundbreaking treatment that cured her rare blood disorder.

U.S. health officials have added two deadly rare diseases to the list of conditions the government recommends states screen ...

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

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

Her 14-year-old twin sons, Grady and Jace, both have PKAN, a rare genetic disorder that affects walking, eating, and speaking ...

Health secretary Robert F. Kennedy Jr. on Tuesday added two deadly rare diseases, Duchenne muscular dystrophy and ...

Addition joins a growing list of launches this year, following in the footsteps of startups like Crystalys Therapeutics and ...

All-RNA, non-viral, LNP-based PRINT™ platform is designed to develop safer, durable, one-time therapies for a broad range of unmet needsAddition is advancing a robust pipeline of PRINTed therapeutics ...

Vanderbilt University Medical Center is recruiting participants for the Phase 3 Hunger Elimination or Reduction Objective ...

With Strand Life Sciences, Reliance is pushing genetic testing toward the mainstream. Incumbents, regulators and doctors are ...

Sol-Gel Technologies (SLGL) fell ~10% in the premarket on Wednesday after the dermatological drug developer announced that ...

Health Canada has approved a new drug treatment for adults living with Fabry disease, a rare genetic disorder that can affect multiple organs and impact the quality of life.