Hemophilia B News and Research

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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

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

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

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

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

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

Nature publishes Sangamo's gene correction strategy for A1AT deficiency

Sangamo BioSciences, Inc. (Nasdaq: SGMO) announced the publication of a preclinical study demonstrating highly specific, functional correction of the alpha 1-antitrypsin (A1AT) gene defect in patient-derived induced pluripotent stem cells (iPSCs) using zinc finger nucleases.

14 Oct 2011

EMA accepts Inspiration's IB1001 MAA for treatment of hemophilia B

Inspiration Biopharmaceuticals, Inc. today announced that the European Medicines Agency (EMA) has validated and accepted the filing of the Marketing Authorization Application (MAA) for Inspiration's IB1001, a recombinant factor IX (FIX) product for the treatment and prevention of bleeding in individuals with hemophilia B.

3 Oct 2011

Express Scripts launches suite of services through HealthBridge Pharma & Biotech

Express Scripts has launched a suite of services for pharmaceutical and biotech companies through HealthBridge Pharma & Biotech, providing a single-solution approach to patient care.

1 Oct 2011

Alnylam commences ALN-PCS Phase I trial in severe hypercholesterolemia

Alnylam Pharmaceuticals, Inc., a leading RNAi therapeutics company, announced today that it has initiated dosing in its Phase I clinical trial with ALN-PCS, an RNAi therapeutic targeting proprotein convertase subtilisin/kexin type 9, or PCSK9, for the treatment of severe hypercholesterolemia.

27 Sep 2011

Positive results from PROLOR Biotech's Factor VIIa-CTP preclinical study on hemophilia

PROLOR Biotech, Inc.,, today reported positive results from a comparative study of its biobetter longer-acting version of the hemophilia drug Factor VIIa (Factor VIIa-CTP) in hemophilic mice.

15 Sep 2011

AMT, NIH enter exclusive license agreement for use of AAV5-based gene therapy vectors

Amsterdam Molecular Therapeutics, a leader in the field of human gene therapy, today announced that it has entered into an exclusive license agreement with the US National Institutes of Health (NIH) for use of adeno-associated virus serotype 5 (AAV5)-based gene therapy vectors for liver and brain indications.

6 Sep 2011