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 ...

Gene editing has the potential to cure thousands of people suffering from devastating rare diseases. But the Nobel ...

A girl diagnosed with a rare brain disease after suddenly becoming unresponsive at home has been "fighting like hell", her ...

The Free Care Fund at UPMC Children's Hospital of Pittsburgh ensures no child is denied medical care. And every year since ...

Charli qualifies for a gene therapy trial in California through the Charlotte and Gwenyth Gray Foundation but the family ...

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

Scientists discovered that certain gene changes allow cells to function even when frataxin, the protein lost in Friedreich’s ...

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

Cure Rare Disease (CRD) announced it has been awarded a $7.4 million grant from the California Institute for Regenerative ...

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