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.

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

Research shows new drug candidate can stop tumor growth in animal models

It's a trick any cat burglar knows: to open a locked door, slide a credit card past the latch. Scientists at The Scripps Research Institute (TSRI) tried a similar strategy when they attempted to disrupt the function of MYC, a cancer regulator thought to be "undruggable."

12 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

TSRI develops accurate structural map of complex molecular machines in cells

A team led by researchers at The Scripps Research Institute (TSRI) has used advanced electron microscopy techniques to determine the first accurate structural map of Mediator, one of the largest and most complex "molecular machines" in cells.

6 Jun 2014

$Nektar reports etirinotecan pegol Phase 2 study results in patients with Avastin-refractory high-grade glioma$

Nektar reports etirinotecan pegol Phase 2 study results in patients with Avastin-refractory high-grade glioma

Nektar Therapeutics announced today new data from an investigator-sponsored Phase 2 study of NKTR-102 (etirinotecan pegol) in patients with Avastin-refractory high-grade glioma conducted at Stanford Cancer Institute under the direction of Lawrence Recht, M.D., Professor of Neurology and Neurosurgery, with co-investigator Seema Nagpal, M.D., Clinical Assistant Professor of Neurology and Neurological Sciences, Stanford School of Medicine.

2 Jun 2014

CIRM grants $5.6 million to develop HIV/AIDS therapeutic using Sangamo's ZFN genome-editing technology

Sangamo BioSciences, Inc. announced that the California Institute for Regenerative Medicine (CIRM) has granted a $5.6 million Strategic Partnership Award to fund clinical studies at City of Hope to develop a potentially curative ZFP Therapeutic for HIV/AIDS based on the application of Sangamo's zinc finger nuclease (ZFN) genome-editing technology in hematopoietic stem/progenitor cells (HSPCs).

31 May 2014

TPP wins $500,000 grant from Biogen Idec to help students start and run own businesses

The Biogen Idec Foundation has awarded a $500,000 grant to The Possible Project (TPP), a non-profit organization that teaches high school students how to start and run their own businesses through a tailored curriculum, and access to technology and to mentors from the local business community.

29 May 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

TSRI researchers discover key proteins involved in one type of DNA repair gone awry

Accumulation of DNA damage can cause aggressive forms of cancer and accelerated aging, so the body's DNA repair mechanisms are normally key to good health. However, in some diseases the DNA repair machinery can become harmful.

23 May 2014

Researchers discover key proteins involved in DNA repair gone awry

22 May 2014

Study led by TSRI scientists likely to influence future designs for HIV vaccines, antibody-based therapies

A vaccine or other therapy directed at a single site on a surface protein of HIV could in principle neutralize nearly all strains of the virus—thanks to the diversity of targets the site presents to the human immune system.

16 May 2014

Scripps Research Institute scientists show AIDS vaccine could work against changeable site on HIV

A vaccine or other therapy directed at a single site on a surface protein of HIV could in principle neutralize nearly all strains of the virus-thanks to the diversity of targets the site presents to the human immune system.

15 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

TSRI scientists discover new HIV target

A team led by scientists at The Scripps Research Institute (TSRI) working with the International AIDS Vaccine Initiative (IAVI) has discovered a new vulnerable site on the HIV virus. The newly identified site can be attacked by human antibodies in a way that neutralizes the infectivity of a wide variety of HIV strains.

25 Apr 2014