ROCTAVIAN: BIOMARIN’S FIRST GENE THERAPY FOR HEMOPHILIA A MAKES HEADWAY

People with hemophilia A have a mutation in the gene responsible for producing factor VIII, a protein that stops bleeding by helping the blood form clots. BioMarin’s new gene therapy for hemophilia A, Roctavian, delivers a functional factor VIII gene to the body to help it produce the clotting factor by itself.

More Info : https://myacare.com/blog/rocta....vian-biomarins-first

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