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.

Baxter to acquire Synovis

Baxter International Inc. and Synovis Life Technologies, Inc. today announced a definitive agreement for Baxter to acquire Synovis, a leading provider of biological and mechanical products for soft tissue repair used in a variety of surgical procedures.

From Baxter International Inc. 14 Dec 2011

Gene therapy for bleeding disorder Hemophilia B

In a landmark study, researchers used gene therapy to successfully treat six patients with severe hemophilia, a blood-clotting disorder. Despite the early nature of the study, it is promising. A single infusion using the new treatment worked in some patients for more than a year, boosting their clotting ability significantly.

12 Dec 2011

Research examines novel treatment techniques for hemophilia B, VTE

Bleeding and clotting disorders affect a large number of patients annually and, while accurate diagnosis is important, timely and effective treatment for these disorders is key, as they have the potential to quickly become life-threatening. Research examining novel treatment techniques for bleeding and clotting disorders will be presented today at the 53rd Annual Meeting of the American Society of Hematology.

12 Dec 2011

Gene therapy can reduce painful bleeding episodes in patients with hemophilia B

Symptoms improved significantly in adults with the bleeding disorder hemophilia B following a single treatment with gene therapy developed by researchers at St. Jude Children's Research Hospital in Memphis and demonstrated to be safe in a clinical trial conducted at the University College London (UCL) in the U.K.

12 Dec 2011

Biogen, Samsung partner to develop and market biosimilars

Samsung and Biogen Idec today announced that they have entered into an agreement to invest $300 million to establish a joint venture to develop, manufacture and market biosimilars. Samsung will take a leading role in the joint venture, with Biogen Idec contributing its expertise in protein engineering and biologics manufacturing.

5 Dec 2011

American Plasma Users Coalition awarded $40,000 advocacy grant from CSL Behring

CSL Behring announced today that it has awarded a $40,000 advocacy grant to the American Plasma Users Coalition (A-PLUS) through its Local Empowerment for Advocacy Development (LEAD) program. LEAD grants support the grassroots advocacy efforts of organizations that help people who use plasma-derived or recombinant therapies to manage rare and serious medical disorders.

3 Dec 2011

FDA approves Mylan's Tranexamic Acid Injection ANDA

Mylan Inc. today announced that its business Mylan Institutional has received final approval from the U.S. Food and Drug Administration for its Abbreviated New Drug Application (ANDA) for Tranexamic Acid Injection, 100 mg/mL, packaged in 1,000 mg/10 mL Single-dose Vials.

3 Dec 2011

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