Acquisition adds high-payload herpes simplex virus (HSV) gene therapy targeting genetic skin diseases to LEO Pharma’s ...

Chiesi Group is taking KalVista Pharmaceuticals under its wing, paying $1.9 billion for the biotech’s oral therapy Ekterly to ...

Danish dermatology specialist Leo Pharma is paying $50 million for U.S. gene writing biotech Replay and its preclinical gene ...

An 11-year-old Big Island boy is battling a series of rare conditions that have left him sick to his stomach.

Mitochondrial disease is a rare and complex genetic disorder that affects people in different ways. There is currently no ...

From decoding hidden genetic mutations to testing novel treatments, scientists are making real progress against rare inherited eye diseases. These breakthroughs could mean earlier diagnoses, targeted ...

Gaucher disease is an underdiagnosed genetic disorder; early detection and treatment can prevent complications and ...

Connecticut prides itself on strong schools, world-class healthcare, and a commitment to caring for its most vulnerable residents. We are a state that values education, innovation, and community. Yet ...

After the diagnosis, Manhasset's Lizz Yeh DiMaiolo and Bobby DiMaiolo founded the Rare Remy Foundation to fund research into ...

Anthony Spina has been battling a rare genetic disease since childhood. His younger brother, Nicholas, is facing the same ...

A nursing shortage made it tough for Isaiah Jones to get 24-hour care. That's when his family discovered his new home: ...