Biomarin Pharmaceutical is looking to sell off rights to Roctavian (valoctocogene roxaparvovec-rvox), its approved gene therapy for hemophilia A. “As we focus on the business units aligned with our strategic priorities, today we are announcing the decision to pursue options to divest Roctavian and remove it from…
I have a friend from middle school who has a special place in my heart. We call each other “friends of a lifetime.” Years can pass without us speaking to each other, but we pick up as if no time has passed at all. I am part of her family…
Several years ago, when I was checking some incisions about a week after I had a surgical procedure, I discovered gobs of red, gelatinous goo. No scabs had formed over the incisions like they were supposed to. I have hemophilia, so thankfully, I made it through the surgery safely,…
Hemophilia A is a rare genetic disorder that affects the blood’s ability to clot properly. It is the most common form of hemophilia, responsible for 80% of all cases.
Mim8 (denecimig) is an antibody therapy being developed to prevent or reduce the frequency of bleeding episodes in people with hemophilia A, with or without neutralizing antibodies (inhibitors).
Hemgenix (etranacogene dezaparvovec), formerly known as AMT-061 and EtranaDez, is a gene therapy given as a one-time intravenous (into-the-vein) infusion to treat bleeding episodes in hemophilia B.
Giroctocogene fitelparvovec, formerly known as SB-525 or PF-07055480, is a gene therapy being developed to reduce the risk of bleeding episodes in people with hemophilia A.
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