Hemophilia A News and Research

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UCSB research: Rapamycin drug, a potential treatment for ADPKD

Research performed at UC Santa Barbara points to the drug rapamycin as a potential treatment for kidney disease. The study builds on past research and shows that studies performed on mice are more likely to translate to humans than previously thought.

27 Jan 2010

Biogen Idec and Swedish Orphan Biovitrum commence dosing in rFIXFc Phase I/IIa trial for hemophilia B

Biogen Idec and Swedish Orphan Biovitrum today announced that the first patient was dosed in a registrational, open-label, multicenter trial designed to evaluate the safety, pharmacokinetics and efficacy of the companies’ long-acting, recombinant Factor IX Fc fusion protein (rFIXFc) in hemophilia B patients.

25 Jan 2010

Ipsen and Inspiration Biopharmaceuticals form strategic partnership

Celtic Pharmaceutical Holdings L.P. ("Celtic Pharma") today announced the formation of a strategic partnership between Ipsen and Inspiration Biopharmaceuticals ("Inspiration") to create a world leading hemophilia franchise, an important milestone for the Celtic Pharma portfolio.

21 Jan 2010

Ipsen enters into partnership agreement with Inspiration Biopharmaceuticals

Ipsen and Inspiration Biopharmaceuticals, Inc. (Inspiration) today announced that they have entered into a partnership to create a world leading hemophilia franchise. The partnership is designed to leverage combined expertise and resources to advance a broad portfolio of recombinant proteins, which address all major hemophilia disorders in a unique way by focusing on two significant unmet needs: wider access to treatment with coagulation factors and treatment for inhibitor complications.

21 Jan 2010

CSL Behring awards four advocacy grants to patient organizations in the U.S.

CSL Behring, a global leader in the plasma-protein biotherapies industry and a subsidiary of CSL Limited, has awarded four advocacy grants totaling $87,000 to patient organizations in the United States through the Local Empowerment for Advocacy Development (LEAD) program.

29 Dec 2009

Tel Aviv University's new Genome Analyzer fuels research dreams and tomorrow's cures

To identify the hemophilia mutation that affected Queen Victoria and her European relatives, scientific detectives used a cutting-edge "deep sequencing tool." Able to trace rare genetic disease mutations, the tool can turn a single laboratory into a fertile genetic research center.

22 Dec 2009

HFA urges American Red Cross to end current labor issues over blood supply

The Hemophilia Federation of America (HFA) expresses its concern regarding the ongoing labor issues involving the Teamsters Local 929 and the American Red Cross of Pennsylvania /New Jersey.

15 Dec 2009

McMaster University launches first online interactive educational program on bleeding disorders

McMaster University, in partnership with the Canadian Hemophilia Society and Bayer HealthCare Pharmaceuticals, has launched Canada's first online, interactive educational program on bleeding disorders. The program, Emergency Management of Bleeding Disorders, is available free to all Canadian health professionals to learn about the signs, symptoms, diagnosis and treatment of bleeding disorders.

10 Dec 2009

Wyeth pledges more than 40 million IUs of factor concentrates to WFH

As part of its continued commitment to hemophilia care around the world, Wyeth, now a part of Pfizer, has pledged more than 40 million international units (IUs) of factor concentrates to the World Federation of Hemophilia (WFH).

3 Dec 2009

NHLBI awards stimulus funds for hemophilia drug research

PTC Therapeutics, Inc. (PTC) today announced the receipt of a $1 million Challenge Grant award from the National Heart, Lung, and Blood Institute (NHLBI) as part of the National Institutes of Health (NIH).

2 Dec 2009

FDA approves additional intended use of Roche's cobas TaqScreen MPX Test

Roche announced today that the United States Food & Drug Administration (FDA) approved an additional intended use for its licensed nucleic acid test, to screen source plasma in pools comprised of up to 96 individual donations. The test, called the cobas® TaqScreen MPX Test for use on the cobas s 201 system, is a qualitative, in-vitro test for the simultaneous, direct detection of the human immunodeficiency virus (HIV-1 Group M RNA, HIV-1 Group O RNA , HIV-2 RNA), hepatitis C virus RNA and hepatitis B virus DNA in human plasma.

From Roche Sequencing and Life Science 2 Dec 2009

Canadians with hemophilia A can use new treatment option, XYNTHA

Canadians with hemophilia A - a rare, inherited blood-clotting disorder - now have a new treatment option with the introduction of XYNTHA(TM), a recombinant factor VIII product for both the control and prevention of bleeding episodes and surgical prophylaxis.

2 Dec 2009

Lysteda tablets to treat menorrhagia approved by the FDA

The U.S. Food and Drug Administration today approved Lysteda tablets (tranexamic acid), the first non-hormonal product cleared to treat heavy menstrual bleeding (menorrhagia). Lysteda works by stabilizing a protein that helps blood to clot.

24 Nov 2009

Amsterdam Molecular Therapeutics provides third-quarter 2009 business updates

Amsterdam Molecular Therapeutics, a leader in the field of human gene therapy, today provides its non-audited business update in compliance with the EU transparency directive. This report summarizes material events and AMT's financial position for the third quarter of 2009.

18 Nov 2009

Lack of hemophilia treatment in the developing world costing lives

When modern medicine finds a way to treat a medical condition, people often think that the problem is solved. But we also have to find ways to get that treatment into the hands of those who need it. For example, new research from North Carolina State University shows that much more needs to be done to help get existing treatment to hemophilia patients in the developing world, and that the current lack of treatment there is costing lives.

16 Nov 2009

Gene therapy saves two children with adrenoleukodystrophy

The ELA association and Zinedine Zidane, its emblematic ambassador, are proud to announce a world premiere: the results regaring the gene therapy in adrenoleukodystrophy conducted in France have just been published in the prestigious journal Science. Two children have been treated and their diseases have been halted. The children are doing well, which is unexpected for a disease destroying the brain in a few months. This discovery opens up treatment perspectives for numerous widespread diseases.

6 Nov 2009

LFB Biotechnologies exercises its option to purchase $12.8 million of stocks in GTC Biotherapeutics

GTC Biotherapeutics, Inc. announced that LFB Biotechnologies, a wholly owned subsidiary of LFB S.A. (Laboratoire français du Fractionnement et des Biotechnologies S.A., Les Ulis, France) and a strategic shareholder in GTC, has exercised its option to purchase $12.8 million of additional convertible preferred stock under the terms described in the financing agreements approved by GTC shareholders in July 2009. This transaction provides $6.4 million of new cash proceeds to GTC.

2 Nov 2009

Novo Nordisk re-launches its changing possibilities in hemophilia campaign

Novo Nordisk Inc. announced the re-launch of its changing possibilities in hemophilia(TM) campaign through a rebranded Web site and a new community on Facebook®. People living with hemophilia face many daily challenges, and Novo Nordisk is committed to helping this community realize that there is also a lifetime of possibilities.

From Novo Nordisk 31 Oct 2009

Phase 2a clinical trial in nonsense mutation hemophilia A and hemophilia B initiated

PTC Therapeutics, Inc. (PTC) today 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).

26 Oct 2009

Medgenics signs $7 million agreement to develop the Biopump technology for Factor VIII

Medgenics is pleased to announce that it has signed a preclinical development and option agreement with a major international biopharmaceutical company that is a market leader in the field of hemophilia.

23 Oct 2009