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 ...

Before a heart attack or heart failure, there are warning signs that are often overlooked or dismissed because they are not ...

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

'He was poisoned.' Toxic fumes on planes blamed for deaths of pilots and crew Actor James Ransone, who starred in 'The Wire,' dies from an apparent suicide at 46 At home known for holiday lights, a ...

Snow rollers are rare because it requires very specific weather and ground conditions for nature to make its own snowballs.

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

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

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

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 ...

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.