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Gene therapy for severe hemophilia is approved by FDA

Por: ABC News Health June 29, 2023

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WASHINGTON -- U.S. officials on Thursday approved drugmaker BioMarin's gene therapy for the most common form of hemophilia, an infused treatment that can significantly reduce dangerous bleeding problems.The Food and Drug Administration approved Roctavian for adult patients with severe cases of hemophilia A, the inherited blood-clotting disorder that can lead to bleeding after minor injuries or scrapes. It's the first gene therapy for... + full article



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