Hemophilia News and Research RSS Feed - Hemophilia News and Research

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.
Biogen receives additional patent protection for MS drug TECFIDERA

Biogen receives additional patent protection for MS drug TECFIDERA

Biogen Idec today announced that the U.S. Patent and Trademark Office has granted U.S. Patent No. 8,399,514, which offers additional protection for TECFIDERA (dimethyl fumarate), the company's oral therapeutic candidate for the treatment of multiple sclerosis. [More]
Alnylam begins dosing in Phase I clinical trial with ALN-TTRsc for treatment of ATTR

Alnylam begins dosing in Phase I clinical trial with ALN-TTRsc for treatment of ATTR

Alnylam Pharmaceuticals a leading RNAi therapeutics company, announced today that it has initiated dosing in its Phase I clinical trial with ALN-TTRsc, an RNAi therapeutic targeting transthyretin (TTR) for the treatment of TTR-mediated amyloidosis (ATTR). [More]
Biogen announces FDA acceptance of rFIXFc BLA for treatment of hemophilia B

Biogen announces FDA acceptance of rFIXFc BLA for treatment of hemophilia B

Biogen Idec announced today that the U.S. Food and Drug Administration (FDA) has accepted the company's Biologics License Application (BLA) for the marketing approval of recombinant factor IX Fc fusion protein (rFIXFc) for the treatment of hemophilia B and granted the company a standard review timeline. [More]

Baxter files FEIBA NF BLA with FDA for prophylactic treatment of hemophilia A or B

Baxter International Inc. today announced that the company has submitted a biologics license application (BLA) supplement to the U.S. Food and Drug Administration (FDA) for the approval of prophylaxis treatment of FEIBA NF [Anti-Inhibitor Coagulant Complex], Nanofiltered and Vapor Heated, in patients with hemophilia A or B and inhibitors. [More]
Research roundup: Patients hesitant to focus on costs; Increase in primary care demand is uneven

Research roundup: Patients hesitant to focus on costs; Increase in primary care demand is uneven

One way to contain health care spending is to get patients involved in weighing costs of treatment options with their doctors. Researchers recruited 211 medically insured participants to assess their willingness to consider costs when choosing treatments. The authors found that participants generally "preferred better care, even when the relative benefit it offered was marginal, and even when told that the second-best choice still met the threshold of 'good enough' care from a clinician's perspective. [More]

FDA approves new vial size for CSL Behring’s Privigen to treat primary immunodeficiency

CSL Behring today announced that the United States Food and Drug Administration has approved a 40 g (400 mL) vial size for Privigen, immune globulin intravenous [human]. [More]

Ipsen, Inspiration Biopharmaceuticals sell IB1001 hemophilia B product to Cangene

Inspiration Biopharmaceuticals and Ipsen today announced the closing of the sale of the proprietary hemophilia B product, IB1001 (recombinant FIX), to Cangene Corporation. [More]
New approach could make gene therapy dramatically more effective for HIV patients

New approach could make gene therapy dramatically more effective for HIV patients

A research team from Case Western Reserve University School of Medicine has discovered an approach that could make gene therapy dramatically more effective for patients. [More]

Medgenics closes public offering of 5,600,000 shares of common stock and warrants

Medgenics, Inc., the developer of a novel platform technology for the sustained production and delivery of therapeutic proteins in patients using their own tissue, announces yesterday's closing of an underwritten public offering of 5,600,000 shares of common stock and Series 2013-A warrants to purchase up to an aggregate of 2,800,000 shares of common stock. [More]
Biogen Idec, Sobi release data from rFVIIIFc and rFIXFc phase 3 trials on hemophilia

Biogen Idec, Sobi release data from rFVIIIFc and rFIXFc phase 3 trials on hemophilia

Biogen Idec and Swedish Orphan Biovitrum (Sobi) released data that confirmed the ability of investigational recombinant factors VIII Fc fusion protein (rFVIIIFc) and IX Fc fusion protein (rFIXFc) to provide long-lasting protection from bleeding with fewer injections than are required with the current standard of care for people with hemophilia. [More]
Sangamo BioSciences fourth quarter revenues increase to $8.9 million

Sangamo BioSciences fourth quarter revenues increase to $8.9 million

Sangamo BioSciences, Inc. today reported fourth quarter and full year 2012 financial results and accomplishments. [More]

Elan agrees to restructure Tysabri collaboration with Biogen Idec

Elan Corporation, plc today announced that it has agreed to restructure the Tysabri® collaboration with Biogen Idec. [More]
Positive top-line results from Biogen Idec’s peginterferon beta-1a Phase 3 trial on RRMS

Positive top-line results from Biogen Idec’s peginterferon beta-1a Phase 3 trial on RRMS

Today Biogen Idec released the primary efficacy analysis and safety data from its Phase 3 pivotal clinical trial, ADVANCE. [More]

Inspiration, Ipsen and Baxter enter Asset Purchase Agreement for OBI-1

Ipsen and Inspiration Biopharmaceuticals Inc. today announced they entered into an Asset Purchase Agreement (APA) whereby Baxter International (Baxter) agrees to acquire the worldwide rights to OBI-1, a recombinant porcine factor VIII (rpFVIII) in development for congenital hemophilia A with inhibitors and acquired hemophilia A, and Ipsen's industrial facility in Milford (Boston, MA). [More]

Penn Medicine to establish new center for treatment and research of blood diseases

Penn Medicine will establish the Philadelphia region's first dedicated center for the treatment and research of blood diseases by combining the expertise of physicians who specialize in the care of blood disorder patients of all ages along with basic science and clinical researchers who are working to advance treatments for these illnesses. [More]
Elan, Biogen Idec submit applications to FDA and EMA for updation of TYSABRI labels

Elan, Biogen Idec submit applications to FDA and EMA for updation of TYSABRI labels

Today Biogen Idec and Elan Corporation, plc announced that they have submitted applications to the U.S. Food and Drug Administration (FDA) and European Medicines Agency (EMA) requesting updates to the TYSABRI (natalizumab) labels. [More]
Researchers uncover genetic causes of von Willebrand disease

Researchers uncover genetic causes of von Willebrand disease

In 2006, the lab of Dr. David Ginsburg at the Life Sciences Institute put a call out for siblings attending the University of Michigan to donate blood for a study of blood-clotting disorders. [More]

Biogen Idec files rFIXFc BLA with FDA for treatment of hemophilia B

Today Biogen Idec announced the company recently submitted a Biologics License Application (BLA) to the U.S. Food and Drug Administration (FDA) for the marketing approval of recombinant factor IX Fc fusion protein (rFIXFc) for the treatment of hemophilia B. [More]

Top-line results from Biogen Idec’s dexpramipexole Phase 3 trial on amyotrophic lateral sclerosis

Today Biogen Idec reported top-line results of EMPOWER, a Phase 3 trial investigating dexpramipexole in people with amyotrophic lateral sclerosis (ALS). [More]

AAV-based gene therapy cocktail may help extend lives of children with Canavan disease

Results of a clinical trial that began in 2001 show that a gene therapy cocktail conveyed into the brain by a molecular special delivery vehicle may help extend the lives of children with Canavan disease, a rare and fatal neurodegenerative disorder. [More]