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Roctavian

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health2 years ago•1 min saved

FDA Approves BioMarin's Gene Therapy for Hemophilia A

The FDA has approved Roctavian, a gene therapy treatment for severe hemophilia A, a rare and potentially fatal blood disorder. The treatment involves a single infusion that inserts missing genes and could eliminate the need for frequent injections. Roctavian is the latest in a series of FDA-approved gene therapies, signaling the potential for gene therapy to revolutionize the treatment of various diseases. However, the high cost of gene therapies remains a significant barrier to widespread use.

via The Washington Post|

#fda #future-of-medicine #gene-therapy

health2 years ago•3 min saved

FDA Approves BioMarin's Gene Therapy for Severe Hemophilia A

The FDA has approved Roctavian, the first gene therapy for adults with severe hemophilia A. Hemophilia A is a rare genetic bleeding disorder caused by a deficiency in clotting factor VIII. Roctavian is a one-time gene therapy administered through intravenous infusion, which increases blood levels of factor VIII to reduce the risk of uncontrolled bleeding. The safety and effectiveness of Roctavian were evaluated in a study of adult men with severe hemophilia A, and it was found to significantly reduce bleeding rates. Common side effects include mild changes in liver function, headache, nausea, and fatigue. The approval of Roctavian provides a new treatment option for patients with severe hemophilia A.

via FDA.gov|

#bleeding-disorder #fda #gene-therapy