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 ...
BioMarin Pharmaceutical has struck a deal to buy fellow biotechnology company Amicus Therapeutics for about $4.8 billion in ...
U.S. health officials have added two deadly rare diseases to the list of conditions the government recommends states screen ...
A girl diagnosed with a rare brain disease after suddenly becoming unresponsive at home has been "fighting like hell", her ...
Rare neurological diseases offer unusually clear models for testing earlier diagnosis, precision treatment and long-term ...
BioMarin is acquiring Amicus Therapeutics for $4.8 billion, merging two rare disease-focused biotechs founded around the turn ...
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 ...
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Bizarre diseases science can't solve
Judge refuses to grant Sarah Palin a new trial in her libel lawsuit against the New York Times Ford stock rises after it ...
Once seen as a last resort, bone marrow transplantation is now being viewed as a potential cure for several rare blood ...
Queen’s University has launched a new online resource to empower unpaid carers supporting their loved ones with a rare ...
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