Hemophilia A News and Research

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Penn Medicine to establish new center for treatment and research of blood diseases

Penn Medicine will establish the Philadelphia region's first dedicated center for the treatment and research of blood diseases by combining the expertise of physicians who specialize in the care of blood disorder patients of all ages along with basic science and clinical researchers who are working to advance treatments for these illnesses.

18 Jan 2013

Researchers uncover genetic causes of von Willebrand disease

In 2006, the lab of Dr. David Ginsburg at the Life Sciences Institute put a call out for siblings attending the University of Michigan to donate blood for a study of blood-clotting disorders.

10 Jan 2013

Biogen Idec files rFIXFc BLA with FDA for treatment of hemophilia B

Today Biogen Idec announced the company recently submitted a Biologics License Application (BLA) to the U.S. Food and Drug Administration (FDA) for the marketing approval of recombinant factor IX Fc fusion protein (rFIXFc) for the treatment of hemophilia B.

7 Jan 2013

AAV-based gene therapy cocktail may help extend lives of children with Canavan disease

Results of a clinical trial that began in 2001 show that a gene therapy cocktail conveyed into the brain by a molecular special delivery vehicle may help extend the lives of children with Canavan disease, a rare and fatal neurodegenerative disorder.

20 Dec 2012

Interim results from Inspiration’s OBI-1 and IB1001 hemophilia clinical study

Inspiration Biopharmaceuticals, Inc. (Inspiration) announced interim clinical results for the company's two lead product candidates, an intravenous recombinant porcine FVIII (OBI-1) in the treatment of serious bleeds in acquired hemophilia A (AHA) and an intravenous recombinant FIX (IB1001) for the treatment and prevention of bleeding in children less than 12 years of age with hemophilia B.

13 Dec 2012

uniQure commences acute intermittent porphyria Phase I clinical trial

uniQure B.V., a leader in the field of human gene therapy, today announced the start of its Phase I clinical trial in acute intermittent porphyria (AIP) with the treatment of the first patient.

11 Dec 2012

Patients with sickle cell disease rely more on emergency room

Patients with sickle cell disease rely more on the emergency room as they move from pediatric to adult health care, according to researchers at Washington University School of Medicine in St. Louis.

10 Dec 2012

Moderna Therapeutics closes $40M in financing

Moderna Therapeutics announced today that it has closed more than $40 million in financing to date, led by Flagship Ventures and private investors, which will be used to advance multiple programs toward clinical stage development.

6 Dec 2012

Bayer completes recruitment of first cohort for BAY 86-6150 Phase II/III study in hemophilia

Bayer's BAY 86-6150 is being investigated in a Phase II/III study called TRUST (TReatment with Unique recombinant rFVIIa STudy), and the recruitment of the first cohort is now complete.

28 Nov 2012

Novo Nordisk launches HemaGo mobile app to help individuals with hemophilia

Novo Nordisk announced today the introduction of HemaGo, a mobile application (app) to help individuals with hemophilia and their caregivers monitor the details of treatment, including medications, dosing, bleed information and the impact of hemophilia on life events.

From Novo Nordisk 28 Nov 2012

Medgenics’ EPODURE Biopump technology for EPO delivery receives U.S. patent

Medgenics, Inc., the developer of BiopumpTM a novel technology for the sustained production and delivery of therapeutic proteins in patients using their own tissue, today announced a patent granted by the U.S. Patent and Trademark Office (USPTO) protecting the use of Medgenics' EPODURE Biopump technology for delivery of erythropoietin (EPO).

22 Nov 2012

New insights into what causes uncontrollable bleeding in hemophilia patients

New insights into what causes uncontrollable bleeding in hemophilia patients are provided in a study published by Cell Press on November 20th in the Biophysical Journal. By revealing that blood clots spread in traveling waves through vessels, the study offers new strategies that could lead to the development of more effective treatments for hemophilia as well as common cardiovascular disorders.

20 Nov 2012

Inspiration, Ipsen receive FDA Fast Track designation for OBI-1 to treat AHA

Ipsen and Inspiration Biopharmaceuticals Inc. today announced that Inspiration has received Fast Track designation from the US Food and Drug Administration (FDA) for OBI-1 in acquired hemophilia A.

20 Nov 2012

Alnylam third quarter 2012 revenues decrease to $16.8 million

Alnylam Pharmaceuticals, Inc., a leading RNAi therapeutics company, today reported its consolidated financial results for the third quarter 2012, and company highlights.

6 Nov 2012

Pfizer Hemophilia donates more than 35M IUs of recombinant factor IX therapy to WFH

Pfizer Hemophilia is donating more than 35 million IUs of its recombinant factor IX therapy to the World Federation of Hemophilia (WFH) to help hemophilia B patients in underserved regions of the world.

24 Oct 2012

Hemophilia bleeding risk ‘low’ for childhood sports

Vigorous physical activity is associated with a moderate but transient bleeding risk for hemophilia, resulting in an overall low risk for bleeds, suggests a study of Australian children.

10 Oct 2012

Physical activity associated with increased risk of bleeding in children with hemophilia

Hemophilia is a bleeding disorder that, if untreated, causes recurrent bleeding into joints. "Vigorous physical activity is thought to increase risk of bleeds in children with hemophilia, but the magnitude of the risk is unknown," according to background information in the article. Information about risks associated with physical activity is needed to help inform decisions regarding participation in physical activity, the authors write.

10 Oct 2012

Positive results from Sobi, Biogen Idec's rFIXFc Phase 3 trial on hemophilia B

Biogen Idec and Swedish Orphan Biovitrum (Sobi) today announced positive results from B-LONG, a clinical study that evaluated a new long-lasting clotting factor candidate in people with hemophilia B. Hemophilia B is a rare inherited disorder that impairs blood coagulation.

27 Sep 2012

Roundup: Ore. insurance CO-OP readies coverage; N.J. Medicaid liabilities hurt state's fiscal outlook; Wis. AG appeals collective bargaining ruling

Independent workers -- artists, journalists, information technology professionals, craftsmen and even those who earn their living buying and selling on eBay -- will soon have the ability to buy health coverage without having to dicker with a privately-run insurance company such as Regence BlueCross BlueShield. ... people can sign up when enrollment opens for the health insurance exchange in October 2013.

20 Sep 2012

Alnylam announces complete results from ALN-RSV01 Phase IIb trial on RSV infection

Alnylam Pharmaceuticals, Inc., a leading RNAi therapeutics company, announced today complete results from its Phase IIb trial with ALN-RSV01 for the treatment of respiratory syncytial virus (RSV) infection in lung transplant patients.

6 Sep 2012