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.

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.

10 Jan 2013

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.

7 Jan 2013

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).

3 Jan 2013

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.

20 Dec 2012

Interim results from Inspiration’s OBI-1 and IB1001 hemophilia clinical study

Inspiration Biopharmaceuticals, Inc. (Inspiration) announced interim clinical results for the company's two lead product candidates, an intravenous recombinant porcine FVIII (OBI-1) in the treatment of serious bleeds in acquired hemophilia A (AHA) and an intravenous recombinant FIX (IB1001) for the treatment and prevention of bleeding in children less than 12 years of age with hemophilia B.

13 Dec 2012

uniQure commences acute intermittent porphyria Phase I clinical trial

uniQure B.V., a leader in the field of human gene therapy, today announced the start of its Phase I clinical trial in acute intermittent porphyria (AIP) with the treatment of the first patient.

11 Dec 2012

Isis, Biogen Idec partner to discover and develop antisense drugs against neuromuscular disorders

Biogen Idec and Isis Pharmaceuticals, Inc. today announced that they have entered into a global collaboration agreement under which the companies will discover and develop antisense drugs against three undisclosed targets to treat neurological or neuromuscular disorders.

11 Dec 2012

Patients with sickle cell disease rely more on emergency room

Patients with sickle cell disease rely more on the emergency room as they move from pediatric to adult health care, according to researchers at Washington University School of Medicine in St. Louis.

10 Dec 2012

Ipsen, Active Biotech complete enrollment in tasquinimod Phase III trial for prostate cancer

Active Biotech and Ipsen today announced that the Phase III clinical trial for tasquinimod, a novel compound for the treatment of prostate cancer, is successfully enrolled with over 1,200 randomized patients as planned in the clinical protocol.

10 Dec 2012

Moderna Therapeutics closes $40M in financing

Moderna Therapeutics announced today that it has closed more than $40 million in financing to date, led by Flagship Ventures and private investors, which will be used to advance multiple programs toward clinical stage development.

6 Dec 2012

Baxter enters definitive agreement to acquire Gambro

Baxter International Inc. has entered into a definitive agreement to acquire Gambro AB, a privately held dialysis product company based in Lund, Sweden, for total consideration of 26.5 billion SEK (approximately $4.0 billion USD at current exchange rates).

From Baxter International Inc. 5 Dec 2012

Bayer completes recruitment of first cohort for BAY 86-6150 Phase II/III study in hemophilia

Bayer's BAY 86-6150 is being investigated in a Phase II/III study called TRUST (TReatment with Unique recombinant rFVIIa STudy), and the recruitment of the first cohort is now complete.

28 Nov 2012

Novo Nordisk launches HemaGo mobile app to help individuals with hemophilia

Novo Nordisk announced today the introduction of HemaGo, a mobile application (app) to help individuals with hemophilia and their caregivers monitor the details of treatment, including medications, dosing, bleed information and the impact of hemophilia on life events.

From Novo Nordisk 28 Nov 2012

Alnylam advances Tuschl II patent estate through the USPTO

Alnylam Pharmaceuticals, Inc., a leading RNAi therapeutics company, announced today that the United States Patent and Trademark Office has issued Notices of Allowance for three patent applications from the company's exclusively held Tuschl II patent estate.

26 Nov 2012

Medgenics’ EPODURE Biopump technology for EPO delivery receives U.S. patent

Medgenics, Inc., the developer of BiopumpTM a novel technology for the sustained production and delivery of therapeutic proteins in patients using their own tissue, today announced a patent granted by the U.S. Patent and Trademark Office (USPTO) protecting the use of Medgenics' EPODURE Biopump technology for delivery of erythropoietin (EPO).

22 Nov 2012

New insights into what causes uncontrollable bleeding in hemophilia patients

New insights into what causes uncontrollable bleeding in hemophilia patients are provided in a study published by Cell Press on November 20th in the Biophysical Journal. By revealing that blood clots spread in traveling waves through vessels, the study offers new strategies that could lead to the development of more effective treatments for hemophilia as well as common cardiovascular disorders.

20 Nov 2012

Inspiration, Ipsen receive FDA Fast Track designation for OBI-1 to treat AHA

Ipsen and Inspiration Biopharmaceuticals Inc. today announced that Inspiration has received Fast Track designation from the US Food and Drug Administration (FDA) for OBI-1 in acquired hemophilia A.

20 Nov 2012

ALN RNAi therapeutic program represents novel approach for treatment of AAT deficiency

Alnylam Pharmaceuticals, Inc., a leading RNAi therapeutics company, announced today that it has presented new pre-clinical data with an RNAi therapeutic targeting alpha-1 antitrypsin (AAT) for the treatment of liver disease associated with AAT deficiency. These data were presented at the 63rd Annual Meeting of the American Association for the Study of Liver Diseases (AASLD, "The Liver Meeting") held November 9-13, 2012 in Boston.

15 Nov 2012

Abbott releases details on phase 3 hepatitis C registrational program

Abbott today released details on its phase 3 hepatitis C registrational program following promising results from its phase 2b clinical trial, known as Aviator, presented at the Annual Meeting of the American Association for the Study of Liver Disease (AASLD) in Boston.

13 Nov 2012

Alnylam third quarter 2012 revenues decrease to $16.8 million

Alnylam Pharmaceuticals, Inc., a leading RNAi therapeutics company, today reported its consolidated financial results for the third quarter 2012, and company highlights.

6 Nov 2012