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

Samuel Sotelo was a regular nine-year-old who loved to play outside and run around, until Halloween, when he began to have ...

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

BioMarin is acquiring Amicus Therapeutics for $4.8 billion, merging two rare disease-focused biotechs founded around the turn ...

A mysterious genetic disease wiped out generations of one family before science uncovered its cause. This talk reveals how a ...

MOG antibody-associated disease (MOGAD) is a rare autoimmune disease of the central nervous system. The blood of patients ...

Ipsen’s oral small molecule has failed to reduce the progression of a genetic disease that gradually turns tendons and ...

Judge refuses to grant Sarah Palin a new trial in her libel lawsuit against the New York Times Ford stock rises after it ...

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

As the FDA unveils a parade of initiatives aimed at accelerating drug development for rare diseases, experts appeal for a consistent approval process that will support and further catalyze momentum.

BioMarin Pharmaceutical said on Friday it would acquire Amicus Therapeutics for about $4.8 billion, in the drugmaker's second deal this year to expand its presence in rare diseases.

University of Sydney marine biologists have identified a devastating combination of coral bleaching and a rare necrotic ...