Hemophilia News and Research

RSS

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.

Enrollment complete in Alnylam's ALN-RSV01 Phase IIb study for RSV infection

Alnylam Pharmaceuticals, Inc., a leading RNAi therapeutics company, announced today that it has completed patient enrollment in its Phase IIb study with ALN-RSV01, an RNAi therapeutic for the treatment of respiratory syncytial virus (RSV) infection.

29 Nov 2011

Inspiration commences enrollment in second OBI-1 Accur8 clinical trial program for congenital hemophilia A

Inspiration Biopharmaceuticals, Inc. today announced the initiation of patient enrollment in the second of two pivotal studies in the Company's OBI-1 Accur8 clinical trial program.

28 Nov 2011

Inspiration initiates second OBI-1 phase III clinical trial in congenital hemophilia A with inhibitors

Ipsen today announced that its partner Inspiration Biopharmaceuticals, Inc. has initiated the treatment of the first patient in the second of two pivotal studies from the OBI-1's Accur8 clinical trial program.

28 Nov 2011

Positive preliminary results from Alnylam's ALN-TTR01 Phase I clinical trial for TTR-mediated amyloidosis

Alnylam Pharmaceuticals, Inc., a leading RNAi therapeutics company, announced today positive preliminary results from its Phase I clinical trial with ALN-TTR01, an RNAi therapeutic targeting transthyretin (TTR) for the treatment of TTR-mediated amyloidosis (ATTR).

21 Nov 2011

$GE Healthcare, Neste Jacobs enter alliance to increase self-sufficiency in blood plasma fractionation$

GE Healthcare, Neste Jacobs enter alliance to increase self-sufficiency in blood plasma fractionation

GE Healthcare, the healthcare business of GE and Neste Jacobs, a leading global expert in plasma fractionation plant engineering design and construction, announced today that the companies have formed a strategic alliance to assist countries worldwide to become self-sufficient in the manufacture of blood plasma products.

21 Nov 2011

EMA grants orphan designation to AMT for hemophilia B gene therapy

Amsterdam Molecular Therapeutics, a leader in the field of human gene therapy, announced today that the European Medicines Agency (EMA) has granted orphan designation for its gene therapy program for the treatment of hemophilia B. Orphan designation in the European Union provides several benefits including 10 years of market exclusivity from product launch and access to the central authorization procedure.

16 Nov 2011

Public health officials should address needs of people with blood disorders

Public health should focus not only on reducing the burden of common diseases but also address the needs of people with blood disorders , experts say in a supplement to December's American Journal of Preventive Medicine.

16 Nov 2011

EPO-secreting engineered vessels could reverse anemia

Patients who rely on recombinant, protein-based drugs must often endure frequent injections, often several times a week, or intravenous therapy. Researchers at Children's Hospital Boston demonstrate the possibility that blood vessels, made from genetically engineered cells, could secrete the drug on demand directly into the bloodstream.

15 Nov 2011

Alnylam announces new data from ALN-TTR01 Phase I trial on TTR-mediated amyloidosis

Alnylam Pharmaceuticals, Inc., a leading RNAi therapeutics company, announced today that it will present new data from its ALN-TTR program at the VIIIth International Symposium on Familial Amyloidotic Polyneuropathy being held in Kumamoto, Japan from November 20-22, 2011.

15 Nov 2011

FDA licenses NYBC's HEMACORD stem cell product

The Food and Drug Administration has licensed HEMACORD for allogeneic hematopoietic (blood-forming) stem cell transplantation, the first such approval of a stem cell product in the world.

11 Nov 2011

HyQ phase III study data in patients with primary immunodeficiencies presented at ACAAI meeting

Baxter International Inc. today announced the presentation of its phase III study data for HyQ, an investigational combination immunoglobulin (IG) product for use in patients with primary immunodeficiencies (PI).

From Baxter International Inc. 7 Nov 2011

Baxter launches NUMETA emulsion for infusion in Europe

Baxter International Inc. announced today the European launch of NUMETA (emulsion for infusion).

From Baxter International Inc. 4 Nov 2011

Gene therapy and stem cell transplantation successfully reverse hemophilia A

For the first time, researchers have combined gene therapy and stem cell transplantation to successfully reverse the severe, crippling bleeding disorder hemophilia A in large animals, opening the door to the development of new therapies for human patients.

3 Nov 2011

FEIBA drug safe and effective for patients with hemophilia A

An international research team led by Dr. Cindy Leissinger of Tulane University School of Medicine, along with Dr. Alessandro Gringeri from the University of Milan, has found that a drug commonly used to treat bleeding events in people with a type of severe hemophilia can also be used to prevent such events from happening in the first place.

3 Nov 2011

GSK, Alnylam collaborate to apply VaxiRNA technology for influenza vaccine production

Alnylam Pharmaceuticals, Inc., a leading RNAi therapeutics company, announced today it has formed a collaboration with GlaxoSmithKline (GSK) focused on Alnylam's VaxiRNATM technology for certain GSK vaccine products, including influenza. VaxiRNA is a new RNAi technology for the enhanced production of viruses used in the manufacture of vaccine products. GSK, a leading vaccine manufacturer, is the first company to form a collaboration accessing this new platform.

2 Nov 2011

Studies on effectiveness of new HCV therapies presented at 76th ACG meeting

Studies reporting on the effectiveness of new therapies for chronic Hepatitis C virus are among the clinical science presented at the American College of Gastroenterology's 76th Annual Scientific Meeting, where investigators also presented findings from an age-based risk assessment and screening intervention for Hepatitis C among Baby Boomers, patients aged 50-65, who saw a gastroenterologist for routine colon cancer screening.

1 Nov 2011

Biogen Idec third quarter revenues increase 11% to $1.3 billion

Biogen Idec Inc., a global biotechnology leader in the discovery, development, manufacturing and commercialization of innovative therapies, today announced its third quarter 2011 results.

28 Oct 2011

Biogen Idec enters global collaboration for oral Syk inhibitor program with Portola

Biogen Idec and Portola Pharmaceuticals, Inc. today announced that they have entered into an exclusive, worldwide collaboration and license agreement under which both companies will develop and commercialize highly selective, novel oral Syk inhibitors for the treatment of various autoimmune and inflammatory diseases, including rheumatoid arthritis (RA) and systemic lupus erythematosus (SLE).

28 Oct 2011

Gilead, Bristol-Myers Squibb to develop and commercialize fixed-dose combination for HIV

Bristol-Myers Squibb Company and Gilead Sciences, Inc. today announced a licensing agreement for Bristol-Myers Squibb to develop and commercialize a fixed-dose combination containing Bristol-Myers Squibb's protease inhibitor REYATAZ (atazanavir sulfate) and Gilead's cobicistat, a pharmacoenhancing or "boosting" agent that increases blood levels of certain HIV medicines to potentially allow for one pill once daily dosing.

27 Oct 2011

Genetically engineered clotting factor shows promise against hemophilia

A genetically engineered clotting factor that controlled hemophilia in an animal study offers a novel potential treatment for human hemophilia and a broad range of other bleeding problems.

24 Oct 2011