Hemophilia A News and Research

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Gene therapy for LCA clears another hurdle: Study

Gene therapy for a severe inherited blindness, which produced dramatic improvements last year in 12 children and young adults who received the treatment in a clinical trial, has cleared another hurdle. The same research team that conducted the human trial now reports that a study in animals has shown that a second injection of genes into the opposite, previously untreated eye is safe and effective, with no signs of interference from unwanted immune reactions following the earlier injection.

4 Mar 2010

Greffex Group signs Strategic Alliance Framework Agreement with UPMC and ISMETT

The Greffex Group of regenerative medicine companies announces the signing of a Strategic Alliance Framework Agreement with UPMC and ISMETT, Italy.

2 Mar 2010

AMT announces full year 2009 results; reports modest decrease in operating expenses

Amsterdam Molecular Therapeutics, a leader in the field of human gene therapy, today reported its results for the year to December 31, 2009.

24 Feb 2010

NCC, Pfizer provide over 10M units of ReFacto Antihemophilic Factor to underserved hemophilia patients

The National Cancer Coalition (NCC) is pleased to announce its recent collaboration with Pfizer, Inc to provide hemophilia medicines to underserved patients in the developing world. To date, NCC has received more than 10 million international units of ReFacto® Antihemophilic Factor (Recombinant), a therapy used for the treatment and prevention of bleeding in people with hemophilia A, to assist patients who otherwise would have little opportunity for adequate treatment. The value of this donation is more than $13 million, and it will help many hundreds of hemophilia patients around the world.

23 Feb 2010

Biogen Idec, Swedish Orphan Biovitrum restructure collaboration agreement for rFVIIIFc and rFIXFc programs

Biogen Idec and Swedish Orphan Biovitrum today announced that they have restructured the collaboration agreement for the companies’ long-acting, recombinant Factor VIII Fc fusion protein (rFVIIIFc) in hemophilia A patients and the recombinant Factor IX Fc fusion protein (rFIXFc) in hemophilia B patients.

18 Feb 2010

Genzyme announces fourth-quarter and full-year 2009 financial results

Genzyme Corp., a diversified, global biotechnology company, today announced fourth-quarter and full year 2009 financial results and provided 2010 guidance that reflects growth across its businesses and a focus on strengthening core areas of the company.

18 Feb 2010

Nebraska laws to protect patients and healthcare providers against unfair prescription insurance practices

Dozens of patients and state and national advocacy and healthcare groups, including Nebraska Medical Association and Nebraska Nurses Association, today urged passage of two precedent-setting consumer protection healthcare bills during a hearing at the Nebraska State Capitol. These laws would be the first in the nation to protect patients and healthcare providers against unfair prescription insurance practices.

17 Feb 2010

Special issue reviews current state of science in fetal therapy

Repairing birth defects in the womb. Inserting a tiny laser into the mother's uterus to seal off an abnormal blood flow and save fetal twins. Advancing the science that may allow doctors to deliver cells or DNA to treat sickle cell anemia and other genetic diseases before birth.

17 Feb 2010

Children's Hospital of Philadelphia awarded two grants to research into cellular therapies for blood disorders

The new decade may herald an era of cell therapy -- treating human diseases by delivering highly specific beneficial cells. In the wake of an NIH decision late last year permitting federally funded researchers to use new lines of human embryonic stem cells, the door has opened more widely to stem cell research.

16 Feb 2010

Biogen Idec reports 2009 fourth quarter and full year earnings

Biogen Idec Inc., a global biotechnology leader in the discovery, development, manufacturing, and commercialization of innovative therapies, today announced its full year and fourth quarter 2009 results.

9 Feb 2010

Ipsen reports consolidated group sales of €1,032.8M for 2009

Ipsen reported today its sales for the fourth quarter and full year 2009.

1 Feb 2010

Baxter International reports net income of $572M for the fourth-quarter of 2009

Baxter International Inc. today reported strong performance for the fourth quarter of 2009, and provided its financial outlook for the first quarter and full-year 2010.

From Baxter International Inc. 28 Jan 2010

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