Why should patients with nonsense mutation Hemophilia A (nmHA) or nonsense mutation Hemophilia B (nmHB) consider participating in the Phase 2a trial?
Patients with severe nmHA or nmHB have less than 1% of the normal Factor VIII or IX. Such low levels can result in bleeding episodes that cause destruction of the joints or damage to other tissues. Bleeding episodes can include bleeding in the brain and can cause severe symptoms (such as stroke). Factor VIII or IX protein concentrates are available to treat HA and HB and can be infused by vein to treat bleeding episodes. However, these drugs require frequent infusions to prevent bleeding. In addition, patients can have medical complications associated with use of catheters for frequent intravenous infusions. By contrast, ataluren is taken by mouth three times a day. Studies in animals with a nonsense mutation in the Factor VIII or IX gene have shown that treatment with ataluren partially restores the production of Factor VIII or IX protein. It is hoped that improvements in Factor VIII or Factor IX will be seen in patients participating in the Phase 2a study. If this happens, it may be
Related Questions
- Why should patients with nonsense mutation Hemophilia A (nmHA) or nonsense mutation Hemophilia B (nmHB) consider participating in the Phase 2a trial?
- Why wasn’t the Delta F508 mutation chosen for the Phase 2 VX-770 clinical trial?
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