Gene therapy delivered by microbubbles shows promise in treating hemophilia B, a hereditary bleeding disorder caused by the lack of blood clotting factor IX, new research suggests. The study, “Ultrasound-targeted hepatic delivery of factor IX in hemophiliac mice,” appeared in the journal Gene Therapy. Hemophilia B affects approximately…
Shire has launched a Phase 3 clinical trial (NCT02585960) with Adynovate, a drug approved to help treat and control bleeding in children and adults with hemophilia A. The trial is currently recruiting participants. Adynovate is an injectable recombinant factor VIII (FVIII), designed using pegylation technology, which allows it to last…
Zepatier (elbasvir/grazoprevir) is an effective and safe treatment for hepatitis C infection in hemophilia patients and others with inherited blood disorders — a group that has been particularly exposed to such infection, according to a Phase 3 trial that examined the drug specifically in this patient group. The study,…
The European Medicines Agency (EMA) will include SPK-9001 — an investigational drug for patients living with hemophilia B —  in Priority Medicines (PRIME), a voluntary European regulatory initiative to support medicines that target an unmet medical need. PRIME aims to optimize development plans and speed up evaluation so that these priority medicines can…
My Life, Our Future (MLOF), a national program founded by leaders in the hemophilia and blood disorder community, marked Feb. 28 — Rare Disease Day — by launching the world’s largest research repository of its kind to researchers and scholars. The program is opening to U.S. scientists and will expand to worldwide scientists in 2018.
A Phase 3 extension trial has confirmed Alprolix’s long-term ability to prevent bleeding episodes in hemophilia B patients, and its safety. Those in the B-YOND extension trial did not develop an immune response against Alprolix. Adults and adolescents were treated for three years, and children under 12 for 18 months. “These results…
Adult hemophilia patients use hospital emergency departments mainly due to cardiovascular events, while pediatric patients most frequently complain of injuries, according to researchers. U.S. emergency department (ED) visits by hemophilia patients had a national estimated cost of $60 million in 2012, the year studied. These observations resulted from the retrospective and…
A new delivery mechanism of mRNA therapy was found to efficiently reduce hemophilia B symptoms by correcting the protein deficiency that characterizes the disease in a mouse study. This potential new method might be a viable alternative for many clotting disorders. The study, “Systemic delivery of Factor IX messenger RNA…
A hemophilia patient has died in a Phase 3 trial evaluating Roche’s  experimental treatment emicizumab (ACE910), raising further concern about its safety. Preliminary indications are that the patient died of a rectal hemorrhage and not emicizumab therapy, however, the company said. The death follows reports of serious blood-clotting events in four…
Psychological distress triggers microscopic changes in regions of hemophiliac children’s brains that deal with emotions and cognition — and the changes show up before abnormal results on neuropsychiatric tests, researchers discovered. The team said larger studies are needed to confirm the links they found between brain changes, the stage of the…
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