FDA Approves First Gene Therapy to Treat Adults with Hemophilia B

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Today, the U.S. Food and Drug Administration approved Hemgenix (etranacogene dezaparvovec), an adeno-associated virus vector-based gene therapy for the treatment of adults with Hemophilia B (congenital Factor IX deficiency) who currently use Factor IX prophylaxis therapy, or have current or historical life-threatening hemorrhage, or have repeated, serious spontaneous bleeding episodes.

United Therapeutics Announces Top Line Data From the EXPEDITE Study of Remodulin Induction Prior to Orenitram Therapy

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United Therapeutics Corporation (Nasdaq: UTHR), a public benefit corporation, today announced that a preliminary analysis of the EXPEDITE study in patients suffering from World Health Organization (WHO) Group 1 pulmonary arterial hypertension (PAH)demonstrated that 79% of patients in the study achieved an Orenitram (treprostinil) extended-release tablet dose of at least 4 mg three times daily (TID), for a 12 mg total daily dose at 16 weeks, after an up to eight week induction treatment period with Remodulin (treprostinil) injection. Detailed study results will be made available through scientific disclosure at upcoming medical conferences and in peer-reviewed publications.