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 ...
Rare neurological diseases offer unusually clear models for testing earlier diagnosis, precision treatment and long-term ...
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 ...
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 ...
Scientists discovered that certain gene changes allow cells to function even when frataxin, the protein lost in Friedreich’s ...
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.
Just two months after reviving its prowess in the idiopathic pulmonary fibrosis (IPF) treatment area with rare lung disease ...
Some results have been hidden because they may be inaccessible to you
Show inaccessible results
Feedback