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 unveil comprehensive portrait of the genome of world's first immortal cell line

A team from the University of Washington has unveiled a comprehensive portrait of the genome of the world's first immortal cell line, known as HeLa. The cell line was derived in 1951 from an aggressive cervical cancer that killed Henrietta Lacks, a 31-year-old African-American tobacco farmer and mother of five - the subject of the 2010 New York Times bestseller, The Immortal Life of Henrietta Lacks.

8 Aug 2013

Scientists identify important molecular signal that guides development of brain

Scientists at The Scripps Research Institute (TSRI) have decoded an important molecular signal that guides the development of a key region of the brain known as the neocortex.

8 Aug 2013

Prothrombin complex concentrates more effective at reversing hemorrhage caused by warfarin

Prothrombin complex concentrates are faster and more effective than fresh frozen plasma at reversing hemorrhage caused by the anti-coagulant warfarin, despite plasma being the most commonly used therapy.

3 Aug 2013

New findings could enable development of effective drugs for addiction with fewer side effects

Scientists from the Florida campus of The Scripps Research Institute have described findings that could enable the development of more effective drugs for addiction with fewer side effects.

3 Aug 2013

Baxter, JW Holdings sign distribution, license agreement for omega 3 parenteral nutrition products

Baxter International Inc. and JW Holdings today announced that they have entered into an exclusive distribution and license agreement for parenteral nutritional products containing a novel formulation of omega 3 lipids.

From Baxter International Inc. 31 Jul 2013

Discovery points to new therapeutic strategy for Parkinson's disease

Biologists at The Scripps Research Institute have made a significant discovery that could lead to a new therapeutic strategy for Parkinson's disease.

25 Jul 2013

Study results suggest reexamination of biological, medical importance of X chromosome

Painstaking new analysis of the genetic sequence of the X chromosome—long perceived as the "female" counterpart to the male-associated Y chromosome—reveals that large portions of the X have evolved to play a specialized role in sperm production.

24 Jul 2013

Biogen Idec announces acceptance of marketing applications for review of PLEGRIDY

Today Biogen Idec announced that U.S. and EU regulatory authorities have accepted the marketing applications for the review of PLEGRIDY (peginterferon beta-1a), the company's pegylated subcutaneous injectable candidate for relapsing forms of multiple sclerosis.

19 Jul 2013

CHOP-led research increases numbers of patients who could be treated with gene therapy

Gene therapy researchers have produced a bioengineered decoy that fools the immune system and prevents it from mistakenly defeating the benefits delivered by a corrective gene.

18 Jul 2013

Alnylam releases positive top-line data from ALN-TTRsc Phase I trial for TTR-mediated amyloidosis

Alnylam Pharmaceuticals, Inc., a leading RNAi therapeutics company, announced today positive top-line results from its ongoing Phase I trial of ALN-TTRsc, a subcutaneously administered RNAi therapeutic targeting the transthyretin gene for the treatment of TTR-mediated amyloidosis.

12 Jul 2013

Bayer starts enrollment in BAY94-9027 Phase II/III trial for hemophilia A in children

Bayer HealthCare today announced the company has started to enroll patients in an international Phase II/III trial to evaluate its investigational compound BAY94-9027 for the treatment of hemophilia A in children.

8 Jul 2013

Data shows individualized regimens can help optimize prophylaxis treatment in hemophilia patients

Baxter International Inc. today announced data featuring ADVATE [Antihemophilic Factor (Recombinant), Plasma/Albumin-Free Method], a leading recombinant treatment available worldwide, during the 24th Annual Congress of the International Society on Thrombosis and Haemostasis (ISTH) in Amsterdam, The Netherlands.

From Baxter International Inc. 5 Jul 2013

Biogen, Sobi present new data supporting clinical, safety profile of ELOCTATE for hemophilia A

Biogen Idec nd Swedish Orphan Biovitrum AB (publ) presented new data that support the clinical and safety profile of their long-lasting recombinant factor VIII candidate ELOCTATE for hemophilia A.

4 Jul 2013

Biogen, Sobi present new findings from Phase 3 trial of ALPROLIX at XXIV ISTH Congress

Biogen Idec and Swedish Orphan Biovitrum AB (publ) today presented new findings for their long-lasting recombinant factor IX candidate ALPROLIX for hemophilia B at the XXIV International Society on Thrombosis and Haemostasis Congress in Amsterdam, The Netherlands.

3 Jul 2013

Alnylam Pharmaceuticals presents new pre-clinical data of ALN-AT3 at ISTH meeting

Alnylam Pharmaceuticals, Inc., a leading RNAi therapeutics company, announced today that it has presented new pre-clinical data from its RNAi therapeutic program for the treatment of hemophilia and rare bleeding disorders.

2 Jul 2013

Alnylam presents new pre-clinical data of ALN-CC5 for treatment of complement-mediated diseases

Alnylam Pharmaceuticals, Inc., a leading RNAi therapeutics company, announced today that it has presented new pre-clinical data with ALN-CC5, an RNAi therapeutic targeting complement component C5.

21 Jun 2013

TSRI scientists find dual inhibitor that attacks two enzymes associated with Parkinson's disease

Scientists from the Florida campus of The Scripps Research Institute have found a compound that could counter Parkinson's disease in two ways at once.

21 Jun 2013

Scientists discover new anti-tuberculosis compound that kills drug-resistant TB bacteria

An international team led by scientists at The Scripps Research Institute (TSRI), the Howard Hughes Medical Institute and Albert Einstein College of Medicine of Yeshiva University has identified a highly promising new anti-tuberculosis compound that attacks the tuberculosis (TB) bacterium in two different ways.

18 Jun 2013

TSRI scientists find emotion-related brain region plays major role in sustaining cocaine addiction

Scientists at The Scripps Research Institute (TSRI) have found evidence that an emotion-related brain region called the central amygdala—whose activity promotes feelings of malaise and unhappiness—plays a major role in sustaining cocaine addiction.

13 Jun 2013

New data on PROLOR Biotech's hGH-CTP and MOD-6030 to be presented at ENDO 2013

PROLOR Biotech, Inc., today announced that the company will present new data on its long-acting human growth hormone (hGH-CTP) in Phase III development for the treatment of growth hormone deficiency and its long acting oxyntomodulin (MOD-6030) in preclinical development for the treatment of obesity and type 2 diabetes at ENDO 2013, the 95th Annual Meeting of the Endocrine Society.