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Article: PTC Therapeutics Initiates Phase 2 Clinical Trial of ataluren (PTC124[R]) in Patients with Hemophilia.
- Article from:
- Blood Weekly
- Article date:
- November 12, 2009
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PTC Therapeutics, Inc. (PTC) announced it is expanding the development of ataluren, an investigational new drug, to a third indication with the initiation of a Phase 2a clinical trial in nonsense mutation hemophilia A (nmHA) and hemophilia B (nmHB). Hemophilia is a rare and debilitating genetic disorder that causes loss of blot clotting proteins and can lead to serious, recurrent bleeding episodes. It is estimated that nonsense mutations account for about 10 to 30 percent of all hemophilia cases (see also Hemophilia A).
The purpose of this multi-center, open-label trial is to evaluate the activity, safety, and pharmacokinetics of ataluren in approximately 24 ...