Hemophilia A News and Research

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Researchers overcome obstacle to using viruses to deliver therapeutic genes

Scientists in The Research Institute at Nationwide Children's Hospital have found a way to overcome one of the biggest obstacles to using viruses to deliver therapeutic genes: how to keep the immune system from neutralizing the virus before it can deliver its genetic payload. In a study published recently in Molecular Therapy, researchers found that giving subjects a treatment to temporarily rid the body of antibodies provides the virus safe passage to targeted cells, allowing it to release a corrective or replacement gene to treat disease.

30 Oct 2013

Biogen Idec Q3 total revenues increase 32% to $1.8 billion

Biogen Idec Inc. today reported third quarter 2013 total revenues of $1.8 billion, an increase of 32% over the third quarter of 2012. Non-GAAP diluted EPS for the third quarter of 2013 were $2.35, an increase of 23% over the third quarter of 2012.

28 Oct 2013

State highlights: 'Habilitation' an essential benefit, but what is it?; Va. gov. candidates differ on mental health system; Calif. pays $2.7M for late hearings

A selection of health policy stories from Virginia, California, Colorado, Missouri, Pennsylvania, Kansas and Texas.

25 Oct 2013

DSM, Sanquin Make Factor IX-Recovering Mab Manufacturing Deal

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24 Oct 2013

New smartphone app for patients with hemophilia and related bleeding disorders

BioRx, a national specialty pharmacy and infusion services provider, announced the launch of its industry's first smartphone app designed for patients with hemophilia and related bleeding disorders.

23 Oct 2013

Catalyst, Isu Abxis To Develop Hemophilia B Candidate

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9 Oct 2013

Alnylam advances Development Candidate for ALN-AS1 for treatment of hepatic porphyrias

Alnylam Pharmaceuticals, Inc. (Nasdaq: ALNY), a leading RNAi therapeutics company, announced today that it is advancing its Development Candidate for ALN-AS1, an RNAi therapeutic targeting aminolevulinate synthase-1 (ALAS-1) for the treatment of hepatic porphyrias including acute intermittent porphyria (AIP).

9 Oct 2013

UTHealth researchers find cause for rare type of bleeding disorder

Fifteen years ago, a hematologist came to Dianna Milewicz, M.D., Ph.D., with a puzzle: Multiple generations of an East Texas family suffered from a moderately severe bleeding disorder, but it wasn't hemophilia.

28 Aug 2013

FDA grants Orphan Drug Designation to Alnylam ALN-AT3 for treatment of hemophilia B

Alnylam Pharmaceuticals, Inc. (Nasdaq: ALNY), a leading RNAi therapeutics company, announced today that the U.S. Food & Drug Administration (FDA) has granted an Orphan Drug Designation to ALN-AT3 as a therapeutic for the treatment of hemophilia B.

17 Aug 2013

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

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

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

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

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

Duke researchers find novel way to repair gene responsible for Duchenne muscular dystrophy

Using a novel genetic 'editing' technique, Duke University biomedical engineers have been able to repair a defect responsible for one of the most common inherited disorders, Duchenne muscular dystrophy, in cell samples from Duchenne patients.

5 Jun 2013

New technique selectively represses unwanted immune reactions without disabling immune system

The human immune system is remarkably efficient, but sometimes its attack is misdirected, leading to allergies, autoimmune diseases and rejection of transplant organs and therapeutic drugs. Current immune suppressants have major drawbacks, but a team from The Scripps Research Institute (TSRI) has demonstrated a new technique that may lead to a better way to selectively repress unwanted immune reactions without disabling the immune system as a whole.

4 Jun 2013