Hemophilia B News and Research

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New book on hemophilia, unintended consequences of medical progress

The Bleeding Disease: Hemophilia and the Unintended Consequences of Medical Progress (John Hopkins University Press) is the new book by NJIT Associate Professor Stephen Pemberton. The book recounts the promising and perilous history of medical and social efforts to manage hemophilia in 20th-century America.

19 Aug 2011

Gene therapy cocktail can treat inherited diseases with 'misfolded' proteins

In a kind of molecular gymnastics, scientists at the University of North Carolina at Chapel Hill School of Medicine have devised a gene therapy cocktail that has the potential to treat some inherited diseases associated with "misfolded" proteins.

16 Aug 2011

Hemophilia Foundation of Michigan announces third annual fundraising walk at the Detroit Zoo

The Hemophilia Foundation of Michigan is pleased to announce: Walkin' on the Wild Side for Hemophilia, its third annual fundraising walk at the Detroit Zoo. Walkin' on the Wild Side for Hemophilia takes place on Sunday, August 28, 2011.

4 Aug 2011

Inspiration presents OBI-1 pivotal study data on hemophilia at 23rd ISTH congress

Ipsen today announced that its partner Inspiration Biopharmaceuticals, Inc. presented data from its clinical development program for OBI-1, an intravenous (IV) recombinant porcine factor VIII product (rpFVIII), intended for the treatment of bleeding in people with hemophilia A with inhibitors and in people with acquired hemophilia.

28 Jul 2011

New preclinical data from Baxter's BAX 499 hemophilia study presented at ISTH

Baxter International Inc. announced today that new data from a series of studies on BAX 499, an investigational compound for potential subcutaneous hemophilia therapy, were presented this week during the 23rd annual congress of the International Society on Thrombosis and Haemostasis (ISTH) in Kyoto, Japan.

From Baxter International Inc. 27 Jul 2011

Biogen Idec reports revenue of $1.2 billion for second quarter 2011

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

26 Jul 2011

Swedish Orphan Biovitrum, Biogen Idec announce data of rFVIIIFc Phase 1/2a trial for hemophilia A

Biogen Idec and Swedish Orphan Biovitrum today announced Phase 1/2a trial data showing that the companies' long-lasting fully-recombinant factor VIII Fc fusion protein (rFVIIIFc) was well tolerated and demonstrated an approximately 1.7-fold increase in half-life compared with Advate, a commercially-available factor VIII product, in 16 previously-treated patients with severe hemophilia A.

26 Jul 2011

CSL Behring supports World Federation of Hemophilia with 1 million IUs of von Willebrand factor/Factor VIII

CSL Behring has donated more than 1 million international units (IU) of von Willebrand factor (VWF)/Factor VIII (FVIII) replacement medication to patients through the World Federation of Hemophilia (WFH). The donation supports WFH's progress in improving the diagnosis and treatment of bleeding disorders in developing countries through its Global Alliance for Progress (GAP) program.

18 Jul 2011

IMM researcher wins prestigious Early Career Bayer Hemophilia Award

Vanessa Oliveira, researcher at the Instituto de Medicina Molecular in Portugal won a prestigious and competitive Early Career Bayer Hemophilia Award. Oliveira will develop a strategy to reprogramme the immune system of hemophilia patients to avoid immune targeting of the most used clotting therapeutics.

14 Jul 2011

In vivo genome editing restores hemostasis in mouse model of blood clotting disorder

Using an innovative gene therapy technique called genome editing that hones in on the precise location of mutated DNA, scientists have treated the blood clotting disorder hemophilia in mice.

27 Jun 2011

AMT receives CHMP opinion on Glybera Marketing Authorisation Application

Amsterdam Molecular Therapeutics, a leader in the field of human gene therapy, today announced that it has received an opinion on its Marketing Authorisation Application (MAA) for Glybera as a potential therapy for Lipoprotein Lipase Deficiency.

24 Jun 2011

European hematologists raise alarm on insufficient research money for blood disorders

European hematologists raise the alarm on the insufficient research money for blood disorders. "Blood cancers kill over 95,000 in the EU every year; the EU must be made aware that more research funds are needed", states Professor Robin Foa, President of the European Hematology Association (EHA) at their annual congress in London.

8 Jun 2011

Increased blood clotting tendency linked to elevated bowel cancer risk

Back in the mid 19th century, a French doctor, Armand Trousseau, discovered a connection between cancer and thrombosis - the formation of often dangerous blood clots that can lead to venous occlusion.

7 Jun 2011

European Commission grants Orphan Drug Designation for CSL Behring's rVIIa-FP to treat hemophilia

CSL Behring announced today that it has been granted Orphan Drug Designations by the European Commission for the development of its recombinant fusion protein linking coagulation factor VIIa with albumin (rVIIa-FP), a novel therapy to treat hemophilia A and hemophilia B patients with inhibitors.

1 Jun 2011

Sangamo announces presentations of ZFP Therapeutics data at ASGCT 2011

Sangamo BioSciences, Inc. announced today that data from clinical, preclinical and research-stage programs focused on the development of zinc finger DNA-binding protein Therapeutics were described in twenty presentations given by Sangamo scientists and collaborators at the 14th Annual Meeting of the American Society of Gene and Cell Therapy.

23 May 2011

CSL-Behring recombinant factor research presented

Advances that CSL Behring is making in developing innovative new technologies to meet the unmet therapeutic needs of patients with hemophilia A and B will be the topic of an oral presentation at an international medical congress on May 22, 2011. CSL Behring is a global biotherapeutics company specializing in plasma-derived and recombinant therapies and is a subsidiary of CSL Limited (ASX:CSL).

19 May 2011

Stem cells from kidneys - World first from Australian scientists

According to Australian scientists, their latest stem cell research breakthrough will bring new treatments for a growing epidemic of chronic kidney disease. They are the first in the world to generate a type of stem cell from human kidney cells. The Melbourne scientists are hoping their discovery will have far-reaching effects in treating the disease.

19 May 2011

EMA agrees to Swedish Orphan Biovitrum, Biogen Idec's pediatric rFIXFc trial in hemophilia

Biogen Idec and Swedish Orphan Biovitrum today announced that the European Medicines Agency's Pediatric Committee has adopted an opinion agreeing to the pediatric investigational plan for the companies' long-lasting, fully-recombinant Factor IX Fc fusion protein.

9 May 2011

Bayer expands sponsorship agreement with World Federation of Hemophilia

Bayer HealthCare Pharmaceuticals and the World Federation of Hemophilia are announcing a three-year extension of Bayer's sponsorship agreement with WFH.

29 Apr 2011

FDA approves Bayer's new Kogenate FS storage option for hemophilia A treatment

Bayer HealthCare Pharmaceuticals announced today that the U.S. Food and Drug Administration has approved a new storage option for Kogenate FS, antihemophilic factor, now allowing the product to be stored at room temperature for up to one year.

26 Apr 2011