Hemophilia News and Research

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Hemophilia is a rare, inherited bleeding disorder in which your blood doesn’t clot normally. If you have hemophilia, you may bleed for a longer time than others after an injury. You also may bleed internally, especially in your knees, ankles, and elbows. This bleeding can damage your organs or tissues and, sometimes, be fatal.

GTC Biotherapeutics regains US commercialization rights to ATryn from Lundbeck

GTC Biotherapeutics, Inc. announced today that it has signed a definitive agreement whereby the Company has regained US commercialization rights to ATryn from Lundbeck, Inc., a wholly owned subsidiary of H. Lundbeck A/S in Denmark.

17 Jul 2010

Second issue of Novo Nordisk BlueSheet highlights information on diabetes

Novo Nordisk Inc., a global healthcare company and leader in diabetes care, today announced the second issue of the Novo Nordisk BlueSheet, a resource for information on diabetes and chronic disease, highlighting key issues in diabetes prevention, detection, treatment and care.

13 Jul 2010

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

Pain Therapeutics modifies strategic alliance with King Pharmaceuticals for REMOXY outside U.S.

Pain Therapeutics, Inc. announced today a modification of its strategic alliance with King Pharmaceuticals, Inc. for REMOXY outside the U.S. The move signals a desire to target Europe's small and growing market for strong pain medications.

28 Jun 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

Phase 2 proof-of-concept data for Novo Nordisk's degludec presented at ADA 2010

Novo Nordisk today presented phase 2 proof-of-concept data for its investigational insulin degludec, an ultra-long-acting basal insulin, which demonstrated the potential to help achieve target glycemic control when used once daily or three times weekly. These data were presented at the 70th Scientific Sessions of the American Diabetes Association.

28 Jun 2010

Data shows Victoza produces greater reductions in A1c, body weight and FPG than Januvia

Late-breaking Novo Nordisk extension data presented today at the 70th Annual Scientific Sessions of the American Diabetes Association showed that once-daily Victoza in combination with metformin produced sustained and significantly greater reductions in A1c, body weight and fasting plasma glucose than Januvia plus metformin at 52 weeks.

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

GTC Biotherapeutics closes new $7 million secured convertible debt financing

GTC Biotherapeutics, Inc. announced the closing of a new $7 million secured convertible debt financing by its strategic partner, LFB Biotechnologies SAS, in connection with a restructuring of GTC's operations which will result in a downsizing of approximately 30 full-time positions at its headquarters and an additional 20 positions at its farm facility.

16 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

CSL Behring granted authorization to market Berinert for HAE in Italy and Luxembourg

CSL Behring announced today it has been granted national marketing authorization in Italy and Luxembourg to market Berinert® for the treatment of acute hereditary angioedema (HAE) attacks in any body location. This latest authorization brings the number of countries to 28 (in Europe, Asia, North America, South America and Australia) in which Berinert is now licensed.

28 May 2010

AMT reports positive preclinical data from cholesterol targeting AAV gene therapy study

Amsterdam Molecular Therapeutics a world leader in gene therapy, today reported positive preclinical data from a study using an AAV gene therapy product to lower cholesterol.

21 May 2010

CSL Behring donates life-saving medicines to assist earthquake victims in Haiti

Direct Relief International announced today that CSL Behring has donated life-saving medicines valued at more than $100,000 to assist earthquake victims in Haiti who are at risk of tetanus or hepatitis B infections. The products will be used in clinics at Hopital Albert Schweitzer in Deschapelles.

14 May 2010

GTC Biotherapeutics reports lower total net loss of $7.7M for first-quarter 2010

GTC Biotherapeutics, Inc. today reported its financial results for the first fiscal quarter ended April 4, 2010. The total net loss for the first quarter was $7.7 million, or $0.26 per share, compared to $10.4 million, or $0.99 per share, for the first quarter of 2009.

12 May 2010

NYBC urges community to donate blood in recognition of Mother's Day

New York Blood Center and its divisions serving the 20 million people of New York City, Long Island, the Hudson Valley, and New Jersey call upon the community to please donate blood in recognition of Mother's Day and families everywhere who are brought together by lives saved through the selfless gifts given by blood donors.

4 May 2010

Hemophilia News and Research

Further Reading

GTC Biotherapeutics regains US commercialization rights to ATryn from Lundbeck

Second issue of Novo Nordisk BlueSheet highlights information on diabetes

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

Pain Therapeutics modifies strategic alliance with King Pharmaceuticals for REMOXY outside U.S.

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

Phase 2 proof-of-concept data for Novo Nordisk's degludec presented at ADA 2010

Data shows Victoza produces greater reductions in A1c, body weight and FPG than Januvia

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

GTC Biotherapeutics closes new $7 million secured convertible debt financing

Researchers underscore role of jumping genes in maintaining genetic diversity

CSL Behring granted authorization to market Berinert for HAE in Italy and Luxembourg

AMT reports positive preclinical data from cholesterol targeting AAV gene therapy study

CSL Behring donates life-saving medicines to assist earthquake victims in Haiti

GTC Biotherapeutics reports lower total net loss of $7.7M for first-quarter 2010

NYBC urges community to donate blood in recognition of Mother's Day

Designing fluidic architecture for scalable diagnostic platforms

Evaluating new approaches for rare disease genomics

Expanding access to NMR with the Fourier 80 Duo

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