Hemophilia A News and Research

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Bayer HealthCare announces over $2.5M for largest awards program in hemophilia

Bayer HealthCare announced today that it awarded more than $2.5 million in funding to 19 recipients from 10 countries through its Bayer Hemophilia Awards Program (BHAP). BHAP is the largest awards program of its kind in hemophilia, providing funds that support clinical and basic research, encourage new physicians to enter the field and enable continuing education for caregivers around the world.

12 Jul 2010

Biogen Idec, Swedish Orphan Biovitrum present results from rFIXFc Phase 1/2a study in hemophilia B

Biogen Idec and Swedish Orphan Biovitrum AB today announced results from a Phase 1/2a open-label, dose-escalation, safety and pharmacokinetic study of the companies' long-lasting, fully-recombinant factor IX Fc fusion protein (rFIXFc) in hemophilia B patients. The data, which were presented at the World Federation of Hemophilia Congress in Buenos Aires, Argentina, on July 11, 2010, showed that rFIXFc was well tolerated and demonstrated an approximately three-fold increase in half-life compared to historical data for existing therapies.

12 Jul 2010

Biogen Idec, Swedish Orphan Biovitrum announce advancement of rFVIIIFc into registrational study

Biogen Idec and Swedish Orphan Biovitrum today announced that they plan to advance the companies' long-lasting, fully-recombinant factor VIII Fc fusion protein (rFVIIIFc) into a registrational clinical trial in people with hemophilia A. The decision to advance the program is based on promising data from a Phase 1/2a open-label, cross-over, multi-center, dose-escalation study that evaluated the safety and pharmacokinetics of an intravenous injection of rFVIIIFc in 16 previously-treated patients with severe hemophilia A.

12 Jul 2010

Pfizer to present results of hemophilia studies at WFH 2010 Congress

Pfizer Inc, the world's leading biopharmaceutical company, today announced that the results of a number of hemophilia studies will be presented at the World Federation of Hemophilia 2010 Congress taking place July 10-14, 2010, in Buenos Aires, Argentina. Key research includes a pre-clinical evaluation of recombinant factor Xa as a potential new approach to restoring hemostasis, as well as a study assessing the potential for an engineered recombinant factor VIIa molecule to improve therapeutic outcomes in mouse models of hemophilia.

9 Jul 2010

Bayer HealthCare to present BAY86-6150 rFVIIa Phase I clinical data at XXIX WFH International Congress

Phase I clinical data for BAY86-6150, a novel recombinant factor VIIa protein (rFVIIa) being developed by Bayer HealthCare, will be presented at the XXIX International Congress of the World Federation of Hemophilia (WFH) to be held in Buenos Aires, Argentina from Saturday, July 10-Wednesday, July 14.

9 Jul 2010

Scientists identify potential disease genes in humans and mice

An international consortium of scientists of Helmholtz Zentrum München and the University of Toronto has identified previously unknown potential disease genes in humans and mice. Genes on the X chromosome, which regulate embryonic development, are the focus of the current publication in the renowned journal Genome Research. Men have only one X chromosome, and therefore mutations on this chromosome disproportionately affect males, frequently leading to serious diseases such as hemophilia, muscular dystrophy and mental retardation.

7 Jul 2010

Transposons create genomic instability and are implicated in cancer and other diseases: Research

Transposons, or "jumping genes," make up roughly half of the human genome. Geneticists previously estimated that they replicate and insert themselves into new locations roughly one in every 20 live births.

28 Jun 2010

Ipsen, Inspiration receive positive opinion in Europe for orphan drug status of OBI-1 in treatment of hemophilia

Ipsen and Inspiration Biopharmaceuticals, Inc. announced today that the Committee for Orphan Medicinal Products of the European Medicines Agency has issued a positive opinion on the granting of orphan drug status for OBI-1 for the treatment of hemophilia.

17 Jun 2010

Researchers underscore role of jumping genes in maintaining genetic diversity

Using high-throughput sequencing to map the locations of a common type of jumping gene within a person's entire genome, researchers at the University of Pennsylvania School of Medicine found extensive variation in these locations among the individuals they studied, further underscoring the role of these errant genes in maintaining genetic diversity.

4 Jun 2010

Some Republican states opting out of high risk health insurance pools

News outlets covered state responses to the federal government's deadline on high risk insurance pools.

3 May 2010

Jumping elements in primates may have important consequences for studies of human genetic diseases

The density of transposable (jumping) elements between sex chromosomes in primates may have important consequences for the studies of human genetic diseases, say Penn State University researchers.

30 Apr 2010

Pain Therapeutics reports net loss of $1.0 million for first-quarter 2010

Pain Therapeutics, Inc., a biopharmaceutical company, today reported first quarter 2010 financial results. Net loss for Q1 2010 was $1.0 million, or $0.02 per share, compared to a net loss of $1.8 million, or $0.04 per share, for Q1 2009.

29 Apr 2010

Pharmacological suppression of nonsense mutations effective in treating genetic defects

Genetic mutation can disrupt the way human cells make proteins, which in turn leads to inherited disease. David Bedwell, a professor in the University of Alabama at Birmingham (UAB) Department of Microbiology, says scientists are closer than ever to producing drugs that fix this disrupted-protein pathway and drastically improving treatment of genetic disease.

27 Apr 2010

Scientists now closer to produce drugs to fix disease-causing mutations, spare healthy genes

Inherited diseases such as cystic fibrosis can be caused by genetic "nonsense mutations" that disrupt the way human cells make proteins. David Bedwell, Ph.D., a professor in the University of Alabama at Birmingham Department of Microbiology, says scientists are now closer to producing drugs that will fix this disruption and drastically improve treatment of genetic disease.

27 Apr 2010

Baxter International's global sales for first-quarter advances by 11% to $3.1 billion

Baxter International Inc. today reported first quarter net income of $525 million, an increase of 2 percent from $516 million reported in the first quarter of 2009. Earnings per diluted share of $0.86 increased 4 percent from $0.83 per diluted share reported in the prior-year period. Baxter's first quarter financial results included a one-time, non-cash special charge of $39 million (or $0.07 per diluted share) related to a change in the tax treatment of post-retirement prescription drug benefits under recent U.S. healthcare reform legislation.

From Baxter International Inc. 22 Apr 2010

TYSABRI drives Biogen Idec's first-quarter revenues of $1.1 billion

Biogen Idec Inc., a global biotechnology leader in the discovery, development, manufacturing, and commercialization of innovative therapies, today announced its first quarter 2010 results.

20 Apr 2010

Plant-based system could help improve lives of hemophilic patients

Hemophilia, a disease linked with legends of European monarchs, frail heirs and one flamboyant charlatan called Rasputin, still afflicts many people today.

31 Mar 2010

AMT initiates dosing in Phase I/II gene therapy clinical trial for hemophilia B

Amsterdam Molecular Therapeutics, a leader in the field of human gene therapy, announced today that the first patient has been dosed in the Phase I/II exploratory clinical trial with a gene therapy product for hemophilia B, a seriously debilitating and potentially lethal disease.

10 Mar 2010

Senators to FDA: Lift ban on gays donating blood

The Associated Press: "The time has come to change a policy that imposes a lifetime ban on donating blood for any man who has had gay sex since 1977, 18 senators said Thursday.

5 Mar 2010

Recipients for 2010 Scholar Awards announced

The American Society of Hematology announces the 2010 recipients of its Scholar Awards. The program is designed to support hematologists who have chosen a career in research by providing partial salary or other support during that critical period required for completion of training and achievement of status as an independent investigator.

5 Mar 2010

Hemophilia A News and Research

Bayer HealthCare announces over $2.5M for largest awards program in hemophilia

Biogen Idec, Swedish Orphan Biovitrum present results from rFIXFc Phase 1/2a study in hemophilia B

Biogen Idec, Swedish Orphan Biovitrum announce advancement of rFVIIIFc into registrational study

Pfizer to present results of hemophilia studies at WFH 2010 Congress

Bayer HealthCare to present BAY86-6150 rFVIIa Phase I clinical data at XXIX WFH International Congress

Scientists identify potential disease genes in humans and mice

Transposons create genomic instability and are implicated in cancer and other diseases: Research

Ipsen, Inspiration receive positive opinion in Europe for orphan drug status of OBI-1 in treatment of hemophilia

Researchers underscore role of jumping genes in maintaining genetic diversity

Some Republican states opting out of high risk health insurance pools

Jumping elements in primates may have important consequences for studies of human genetic diseases

Pain Therapeutics reports net loss of $1.0 million for first-quarter 2010

Pharmacological suppression of nonsense mutations effective in treating genetic defects

Scientists now closer to produce drugs to fix disease-causing mutations, spare healthy genes

Baxter International's global sales for first-quarter advances by 11% to $3.1 billion

TYSABRI drives Biogen Idec's first-quarter revenues of $1.1 billion

Plant-based system could help improve lives of hemophilic patients

AMT initiates dosing in Phase I/II gene therapy clinical trial for hemophilia B

Senators to FDA: Lift ban on gays donating blood

Recipients for 2010 Scholar Awards announced

Achieving and maintaining ISO/IEC 27001 and BS 10012 certifications

Expanding research and clinical options for children with cancer

Examining how pain could play a direct protective role in the gut

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