Hemophilia B News and Research

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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

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

Hemophilia B News and Research

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

Gene therapy for LCA clears another hurdle: Study

Greffex Group signs Strategic Alliance Framework Agreement with UPMC and ISMETT

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

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

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

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

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

Special issue reviews current state of science in fetal therapy

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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