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.

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

Medgenics second quarter net loss increases to $2.32 million

Medgenics, Inc., the developer of a novel technology for the sustained production and delivery of therapeutic proteins in patients using their own tissue, today reported financial results for the three and six months ended June 30, 2011.

12 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

Baxter institutes Baxter Ventures to invest $200M in promising early-stage therapies

Baxter International Inc. announced today that it has established Baxter Ventures to invest up to $200 million in equity in promising early-stage companies developing therapies that complement Baxter's existing portfolio.

From Baxter International Inc. 30 Jul 2011

PPTA applauds introduction of 'Preserving Access to Orphan Drugs Act'

The Plasma Protein Therapeutics Association (PPTA) applauds the introduction of bipartisan legislation, the Preserving Access to Orphan Drugs Act (H.R. 2672) and (S. 1423), which will safeguard the development of drugs and therapies that treat patients with rare diseases by eliminating barriers to innovation.

30 Jul 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

Puget Sound Blood Center, Seattle Children's inaugurate new on-site lab

Today, Seattle Children's and Puget Sound Blood Center opened a new blood laboratory to serve Children's patients exclusively. Located within Seattle Children's main campus and staffed by Puget Sound Blood Center employees 24 hours every day of the year, the facility will bring the lab closer to the patient.

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

Biogen Idec's FAMPYRA receives EU conditional approval for treating functional impairment in MS

Biogen Idec announced today it has received conditional approval from the European Commission for FAMPYRA® (prolonged-release fampridine tablets) to improve walking in adult patients with multiple sclerosis who have walking disability.

25 Jul 2011

Baxter GAMMAGARD LIQUID for PI receives FDA approval for subcutaneous administration

Baxter International Inc. announced today that the U.S. Food and Drug Administration has approved the subcutaneous administration of GAMMAGARD LIQUID 10% [Immune Globulin Infusion (Human)] for patients with primary immunodeficiency.

From Baxter International Inc. 25 Jul 2011

Baxter net income increases 15% to $615 million for second quarter 2011

Baxter International Inc. today announced strong financial results for the second quarter of 2011, and raised its financial outlook for full-year 2011.

From Baxter International Inc. 21 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

Ipsen, Salk Institute renew Life Sciences Program

Ipsen and The Salk Institute for Biological Studies announced today that they are renewing the Ipsen Life Sciences Program at The Salk Institute.

12 Jul 2011

IGR, IPSEN sign 3 year medical oncology agreement

IPSEN, a global biopharmaceutical company represented by Claude Bertrand, Executive Vice President, R&D and Chief Scientific Officer, and Institut de cancérologie Gustave Roussy, a leading international cancer center represented by Professor Alexander Eggermont, General Director, today announced the signature of a partnership in the area of medical oncology to leverage the combined expertises of their respective R&D teams.

12 Jul 2011

Top-line results from HyQ subcutaneous IG phase III study in patients with primary immunodeficiency

Baxter International Inc. and Halozyme Therapeutics, Inc. today announced top-line results of a phase III study of HyQ, an investigational facilitated subcutaneous immune globulin (IG) product for use in patients with primary immunodeficiency (PI).

From Baxter International Inc. 8 Jul 2011

Glybera gene therapy reduces pancreatitis risk in Lipoprotein Lipase Deficiency patients

Amsterdam Molecular Therapeutics, a leader in the field of human gene therapy, today released data showing that its gene therapy Glybera® (alipogene tiparvovec) significantly reduces the risk of pancreatitis in patients with Lipoprotein Lipase Deficiency (LPLD). Pancreatitis, or inflammation of the pancreas, is a major clinical symptom of LPLD.

29 Jun 2011

Tibotec, Gilead Sciences enter license agreement for new combination product of PREZISTA and cobicistat

Tibotec Pharmaceuticals today announced that it has entered into a license agreement with Gilead Sciences, Inc., for the development and commercialization of a new once-daily single tablet fixed-dose antiretroviral combination product containing Tibotec's protease inhibitor PREZISTA® (darunavir) and Gilead's cobicistat, an investigational pharmacoenhancing or "boosting" agent.

28 Jun 2011