Hemophilia A News and Research

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New injectable polymer could strengthen blood clots

Most military battlefield casualties die before ever reaching a surgical hospital. Of those soldiers who might potentially survive, most die from uncontrolled bleeding.

11 Mar 2015

Finding may help advance experimental approach to improving public health

Scientists have discovered a new way to manipulate how cells function, a finding that might help advance an experimental approach to improving public health: DNA vaccines, which could be more efficient, less expensive and easier to store than traditional vaccines.

5 Mar 2015

Bayer announces FDA acceptance of BAY 81-8973 BLA for treatment of hemophilia A

Bayer HealthCare today announced that the U.S. Food and Drug Administration has accepted the company's Biologics License Application for BAY 81-8973, a recombinant Factor VIII compound. Bayer is seeking FDA approval of the investigational compound, proposed trade name Kovaltry, for the treatment of hemophilia A in children and adults.

4 Mar 2015

CSL Behring donates protein therapies to World Federation of Hemophilia

In recognition of Rare Disease Day and as part of its ongoing commitment to the global bleeding disorders community, CSL Behring announced today that it is donating 2 million international units (IUs) of protein therapies to the World Federation of Hemophilia.

28 Feb 2015

Spark Therapeutics, Pfizer partner to develop SPK-FIX for potential treatment of hemophilia B

Spark Therapeutics, a late-stage gene therapy company developing treatments for debilitating genetic diseases, announced today that it has entered into a global collaboration with Pfizer Inc. for the development and potential commercialization of SPK-FIX, a development program advancing proprietary, bio-engineered adeno-associated virus (AAV) vectors for the potential treatment of hemophilia B.

8 Dec 2014

Baxter seeks FDA approval of BAX 855 for treatment of people with hemophilia A

Nektar Therapeutics reported that partner Baxter International Inc. today announced that the company has submitted a biologics license application (BLA) to the United States Food and Drug Administration for the approval of BAX 855, an investigational, extended half-life recombinant factor VIII (rFVIII) treatment for hemophilia A based on ADVATE [Antihemophilic Factor (Recombinant)].

From Baxter International Inc. 1 Dec 2014

Gene therapy transforms life for men with severe form of hemophilia B

Gene therapy developed at St. Jude Children's Research Hospital, University College London and the Royal Free Hospital has transformed life for men with a severe form of hemophilia B by providing a safe, reliable source of the blood clotting protein Factor IX that has allowed some to adopt a more active lifestyle, researchers reported.

19 Nov 2014

School nurses reach 98% of students in U.S. public schools to diagnose primary immunodeficiency

School nurses reach 98 percent of the 50,000,000 students in U.S. public schools, grades k-12, and are uniquely positioned to facilitate the early diagnosis of serious medical conditions such as primary immunodeficiency (PI).

28 Aug 2014

Baxter reports positive results from BAX 855 Phase 3 trial for hemophilia A

Nektar Therapeutics reports Baxter International Inc. today announced positive results from its Phase 3 pivotal clinical trial of BAX 855, an investigational, extended half-life recombinant factor VIII (rFVIII) treatment for hemophilia A based on ADVATE [Antihemophilic Factor (Recombinant)], which met its primary endpoint in reducing annualized bleed rates (ABR) in the prophylaxis arm compared to the on-demand arm.

From Baxter International Inc. 22 Aug 2014

Trinidad & Tobago Ministry of Health approves hepatitis C drug telaprevir

The Chemistry, Food & Drugs Division of the Trinidad & Tobago Ministry of Health approved the introduction of Telaprevir to the market, an innovative therapy that significantly increases the cure rate from Hepatitis C, and it is indicated for patients infected with the genotype 1 virus. Telaprevir is available in Trinidad under the trade brand INCIVO®.

26 Jul 2014

Scientists discover new compounds that could alter circadian rhythm

Scientists from the Florida campus of The Scripps Research Institute (TSRI) have discovered a surprising new role for a pair of compounds—which have the potential to alter circadian rhythm, the complex physiological process that responds to a 24-hour cycle of light and dark and is present in most living things.

8 Jul 2014

Bayer, Dimension Therapeutics partner to develop new gene therapy for hemophilia A

Dimension Therapeutics, a company focused on developing novel adeno-associated virus (AAV) gene therapy treatments for rare diseases, today announced it has entered into a collaboration with Bayer HealthCare (Bayer) for the development and commercialization of a novel gene therapy for the treatment of hemophilia A.

23 Jun 2014

Fragile Y hypothesis explains the factors behind chromosome loss

A UT Arlington research team says their study of genetic information from more than 4,000 beetle species has yielded a new theory about why some species lose their Y chromosome and others, such as humans, hang on to it.

18 Jun 2014

Leading experts from key sectors discuss challenges in 340B Drug Discount Program

Leading health care policy experts from community health centers, patient and clinician specialty groups, industry organizations and academic institutions convened yesterday to discuss challenges in the federal 340B Drug Discount Program, and the importance of preserving aspects that truly serve vulnerable, uninsured and underinsured patients.

12 Jun 2014

Alnylam announces new pre-clinical results with DC for ALN-CC5 to treat complement-mediated diseases

Alnylam Pharmaceuticals, Inc., a leading RNAi therapeutics company, announced today new pre-clinical results with its Development Candidate (DC) for ALN-CC5, a subcutaneously administered investigational RNAi therapeutic targeting complement component C5 in development for the treatment of complement-mediated diseases.

10 Jun 2014

Half-life extension technology: an interview with Dermot Pearson, Novozymes Biopharma

A drug's half-life indicates how long a substance will remain active in the body. It is essentially the period of time that it takes for the concentration of the amount of the drug to be reduced by half, and therefore, determines how frequently a drug needs to be administered to maintain its therapeutic effect.

27 May 2014

FDA approves Bayer's Kogenate FS antihemophilic factor VIII to prevent bleeding episodes in adults with hemophilia A

Bayer HealthCare announced today that the U.S. Food and Drug Administration (FDA) has approved a new indication for Bayer's Kogenate® FS antihemophilic factor VIII (recombinant), for routine prophylaxis to prevent or reduce the frequency of bleeding episodes in adults with hemophilia A.

12 May 2014

Voluntis closes €20.75 million ($29 million) Series D financing round

Voluntis, a company pioneering therapeutic companion software, today announced the completion of its Series D financing round. With a total of €20.75 million ($29 million), this fund raising is one of the largest rounds of investment in the m-health sector this year.

28 Apr 2014

Biogen Idec's revenue increases 51% to $2.1 billion in first quarter 2014

Biogen Idec Inc. today reported first quarter 2014 results, including revenue of $2.1 billion, a 51% increase compared to the first quarter of 2013.

23 Apr 2014

New gene-editing system holds potential for treating many genetic disorders

Using a new gene-editing system based on bacterial proteins, MIT researchers have cured mice of a rare liver disorder caused by a single genetic mutation.

31 Mar 2014