U.S. health officials have added two deadly rare diseases to the list of conditions the government recommends states screen ...
The FDA has issued its stamp of approval to a new, cell-based option to treat Wiskott-Aldrich syndrome (WAS), marking the ...
BioMarin is acquiring Amicus Therapeutics for $4.8 billion, merging two rare disease-focused biotechs founded around the turn ...
BioMarin Pharmaceutical said on Friday it would acquire Amicus Therapeutics for about $4.8 billion, in the drugmaker's second ...
Danish pharmaceutical multinational Novo Nordisk is expanding its presence in inherited blood disorder treatments ...
The seven-year medical odyssey that once defined rare disease diagnosis is being rewritten by algorithms capable of identifying medical zebras in the time it takes to refresh your social media feed.
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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 ...
More than 500 European patients with previously undiagnosed rare disorders received a genetic diagnosis through research carried out by an extensive European collaboration, the Solve-Rare Diseases ...
Backed by a handful of prominent investors including the Gates Foundation, the startup is pursuing an approach it believes ...
San Rafael-based BioMarin is putting down a hefty all-cash wager on the rare-disease market, agreeing to buy New Jersey ...
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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 ...
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