BioMarin Pharmaceutical has struck a deal to buy fellow biotechnology company Amicus Therapeutics for about $4.8 billion in ...
Ipsen’s oral small molecule has failed to reduce the progression of a genetic disease that gradually turns tendons and ...
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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 ...
BioMarin is acquiring Amicus Therapeutics for $4.8 billion, merging two rare disease-focused biotechs founded around the turn ...
San Rafael-based BioMarin is putting down a hefty all-cash wager on the rare-disease market, agreeing to buy New Jersey ...
U.S. health officials have added two deadly rare diseases to the list of conditions the government recommends states screen ...
As the FDA unveils a parade of initiatives aimed at accelerating drug development for rare diseases, experts appeal for a ...
Gene editing has the potential to cure thousands of people suffering from devastating rare diseases. But the Nobel ...
The effects allowed these minors who could sit but not stand to move like they've never done before, including walking and ...
Soleo Health has appointed Jody Thompson as senior vice president of business development, rare disease, to broaden the company’s portfolio of exclusive and limited distribution therapies for rare, ...
Backed by Italy-based Fondazione Telethon ETS, Waskyra, for Wiskott-Aldrich syndrome, is the first gene therapy from a ...
Rare neurological diseases offer unusually clear models for testing earlier diagnosis, precision treatment and long-term ...
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