Hemophilia B News and Research

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New data on extending the serum half-life of albumin in vivo announced by Novozymes Biopharma

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27 Jan 2014

Study shows wide-ranging variation in patterns of X chromosome inactivation in female mice

Producing brightly speckled red and green snapshots of many different tissues, Johns Hopkins researchers have color-coded cells in female mice to display which of their two X chromosomes has been made inactive, or “silenced.”

10 Jan 2014

FDA approves Baxter's FEIBA for prophylactic treatment of hemophilia patients with inhibitors

Baxter International Inc. today announced that the United States Food and Drug Administration granted approval of Baxter's FEIBA [Anti-Inhibitor Coagulant Complex], the first and only FDA-approved treatment for routine prophylaxis to prevent or reduce the frequency of bleeding episodes in patients with hemophilia A or B who have developed inhibitors.

From Baxter International Inc. 20 Dec 2013

CHOP scientist receives 2013 E. Donnall Thomas Prize for scientific, clinical research in hemophilia

Katherine A. High, M.D., of The Children's Hospital of Philadelphia (CHOP) was honored for her trailblazing scientific and clinical research in the bleeding disorder hemophilia when she received the 2013 E. Donnall Thomas Prize from the American Society of Hematology (ASH) at its annual meeting on Dec. 9 in New Orleans.

15 Dec 2013

CHOP scientist receives 2013 E. Donnall Thomas Prize for research in bleeding disorder hemophilia

Katherine A. High, M.D., of The Children's Hospital of Philadelphia was honored for her trailblazing scientific and clinical research in the bleeding disorder hemophilia when she received the 2013 E. Donnall Thomas Prize from the American Society of Hematology at its annual meeting on Dec. 9 in New Orleans.

14 Dec 2013

Researchers discover new therapeutic approach for hemophilia

Researchers at the UNC School of Medicine and the Medical College of Wisconsin found that a new kind of gene therapy led to a dramatic decline in bleeding events in dogs with naturally occurring hemophilia A, a serious and costly bleeding condition that affects about 50,000 people in the United States and millions more around the world.

12 Dec 2013

Understanding long-term clinical stability in hemophilia patients living with HIV

People with the genetic blood clotting disorder hemophilia who have been infected with HIV for decades have an increased proportion of immune cells in their blood that specifically target HIV. This protective immune response helps chronically infected hemophilia patients survive, even during periods of HIV activity, according to a study published in BioResearch Open Access, a peer-reviewed journal from Mary Ann Liebert, Inc., publishers.

12 Dec 2013

Baxter seeks FDA approval of OBI-1 for adults with acquired hemophilia A

Baxter International Inc. today announced that the company has submitted a biologics license application (BLA) to the U.S. Food and Drug Administration (FDA) for the approval of OBI-1, a recombinant antihemophilic porcine sequence factor VIII, in patients with acquired hemophilia A.

From Baxter International Inc. 11 Dec 2013

Alnylam presents new pre-clinical data with ALN-AT3 for treatment of hemophilia and RBD

Alnylam Pharmaceuticals, Inc., a leading RNAi therapeutics company, announced today that it has presented new pre-clinical data with ALN-AT3, a subcutaneously administered RNAi therapeutic targeting antithrombin (AT) for the treatment of hemophilia and rare bleeding disorders (RBD), at the 55th Annual Meeting of the American Society of Hematology (ASH) held December 7 - 10, 2013 in New Orleans.

10 Dec 2013

Biotest, EpiVax partner to develop novel, non-immunogenic Factor VIII for hemophilia treatment

Biotest AG, Dreieich, Germany and EpiVax, Inc., Providence, Rhode Island, USA are pleased to announce a new Collaborative Research Agreement. With this collaboration a novel, non-immunogenic Factor VIII should be developed.

3 Dec 2013

FDA extends initial PDUFA date for Biogen Idec's review of ALPROLIX BLA

Biogen Idec announced today that the U.S. Food and Drug Administration has extended the initial Prescription Drug User Fee Act date for its review of the Biologics License Application (BLA) for ALPROLIX, the company's investigational long-lasting recombinant factor IX Fc fusion protein candidate for hemophilia B.

2 Dec 2013

Human Treg responses allow sustained recombinant adeno-associated virus-mediated transgene expression

Successful gene therapy is based on the effective delivery and maintained expression of healthy copies of a gene into tissues of individuals with a disease-associated genetic mutation.

16 Nov 2013

Researchers overcome obstacle to using viruses to deliver therapeutic genes

Scientists in The Research Institute at Nationwide Children's Hospital have found a way to overcome one of the biggest obstacles to using viruses to deliver therapeutic genes: how to keep the immune system from neutralizing the virus before it can deliver its genetic payload. In a study published recently in Molecular Therapy, researchers found that giving subjects a treatment to temporarily rid the body of antibodies provides the virus safe passage to targeted cells, allowing it to release a corrective or replacement gene to treat disease.

30 Oct 2013

Biogen Idec Q3 total revenues increase 32% to $1.8 billion

Biogen Idec Inc. today reported third quarter 2013 total revenues of $1.8 billion, an increase of 32% over the third quarter of 2012. Non-GAAP diluted EPS for the third quarter of 2013 were $2.35, an increase of 23% over the third quarter of 2012.

28 Oct 2013

State highlights: 'Habilitation' an essential benefit, but what is it?; Va. gov. candidates differ on mental health system; Calif. pays $2.7M for late hearings

A selection of health policy stories from Virginia, California, Colorado, Missouri, Pennsylvania, Kansas and Texas.

25 Oct 2013

DSM, Sanquin Make Factor IX-Recovering Mab Manufacturing Deal

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24 Oct 2013

New smartphone app for patients with hemophilia and related bleeding disorders

BioRx, a national specialty pharmacy and infusion services provider, announced the launch of its industry's first smartphone app designed for patients with hemophilia and related bleeding disorders.

23 Oct 2013

Catalyst, Isu Abxis To Develop Hemophilia B Candidate

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9 Oct 2013

Alnylam advances Development Candidate for ALN-AS1 for treatment of hepatic porphyrias

Alnylam Pharmaceuticals, Inc. (Nasdaq: ALNY), a leading RNAi therapeutics company, announced today that it is advancing its Development Candidate for ALN-AS1, an RNAi therapeutic targeting aminolevulinate synthase-1 (ALAS-1) for the treatment of hepatic porphyrias including acute intermittent porphyria (AIP).