FDA Approves BioMarin's Gene Therapy for Hemophilia A
TL;DR
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.
- What is gene therapy, and how does it work? The Washington Post
- FDA OKs companion Dx with BioMarin hemophilia gene therapy FierceBiotech
- BioMarin (BMRN) Gets FDA Nod for Hemophilia A Gene Therapy Yahoo Finance
- FDA Approves BioMartin's Roctavian for Treatment of Severe Hemophilia A Pharmacy Times
- Pharmalittle: FDA approves BioMarin gene therapy for hemophilia A; former Pfizer worker charged with insider trading STAT
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