BioMarin is acquiring Amicus Therapeutics for $4.8 billion, merging two rare disease-focused biotechs founded around the turn ...
A multicenter study led by UC Davis Health has tested a new treatment designed to improve care for people with a rare liver ...
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Scientist diagnosed with the rare disease that took her mother's life dedicates her life to finding a cure
Sonia Vallabh had already obtained her law degree from Harvard, but after her diagnosis, decided to retrain in biomedical ...
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 ...
Backed by Italy-based Fondazione Telethon ETS, Waskyra, for Wiskott-Aldrich syndrome, is the first gene therapy from a ...
A girl diagnosed with a rare brain disease after suddenly becoming unresponsive at home has been "fighting like hell", her ...
U.S. health officials have added two deadly rare diseases to the list of conditions the government recommends states screen ...
The Covid-19 pandemic brought rare disease research to a standstill. Five years later, patients and advocates say the ...
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Lee meets with rare disease-stricken patients
Lee meets with rare disease-stricken patients President Lee Jae Myung (rear, 5th from L), alongside his wife, Kim Hea Kyung ...
For Americans living with rare diseases, the Rare Pediatric Disease Priority Review Voucher Program is a vital tool. Yet its reauthorization is at risk.
Rare diseases are defined as conditions affecting fewer than 200,000 people in the US or less than 1 in 2,000 in Europe. 1-2 While each disease is individually rare, collectively, they represent a ...
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