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.

Novo Nordisk reports positive data from Novoeight phase 3 study in people with haemophilia A

Today, Novo Nordisk announced a new analysis of phase 3 data demonstrating people with haemophilia A who had the highest annualised bleeding rate (ABR) during initial treatment with Novoeight (Antihemophilic Factor [Recombinant]) showed the largest reduction in bleeding over the duration of treatment.

From Novo Nordisk 7 Dec 2014

BRI signs license agreement with SOBI for Kineret (anakinra)

Baylor Research Institute, the research arm of the Baylor Scott & White Health, announced that it has signed an agreement with Swedish Orphan Biovitrum AB (Sobi) to non-exclusively license Baylor's patents pertaining to the treatment of Systemic Onset Juvenile Idiopathic Arthritis (SJIA or SOJIA) using interleukin-1 (IL-1) beta antagonists.

6 Dec 2014

TSRI scientists identify 'mechanoreceptor' protein that mediates sense of touch in mammals

A team led by biologists at The Scripps Research Institute has solved a long-standing mystery in neuroscience by identifying the "mechanoreceptor" protein that mediates the sense of touch in mammals.

5 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

Novo Nordisk announces re-launch of online patient support platform for people with diabetes

Novo Nordisk, a world leader in diabetes care, today announced the re-launch of Cornerstones4Care, an award-winning online patient support platform for people living with diabetes.

From Novo Nordisk 5 Nov 2014

TSRI, STSI researchers receive NIH grants to set up platforms to mine biomedical data

San Diego researchers at The Scripps Research Institute and Scripps Translational Science Institute will receive more than $4.4 million as part of a National Institutes of Health initiative called "Big Data to Knowledge" (BD2K).

10 Oct 2014

CSL Behring strengthens global manufacturing capabilities to meet demand for lifesaving therapies

CSL Behring today announced a multi-year, $450 million two-site global capacity expansion – the latest in a series of recent production expansions to meet the growing need for its lifesaving and life-improving therapies that are used to treat patients with rare and serious medical disorders around the world.

8 Oct 2014

TSRI scientists awarded DARPA grant to build artificial immune system

Scientists from both campuses of The Scripps Research Institute have been awarded a total of $7.9 million from the Defense Advanced Research Projects Agency of the U.S. Department of Defense.

25 Sep 2014

TSRI scientists devise new vancomycin-based antibiotic to rout resistant bacteria

Scientists at The Scripps Research Institute have devised a new antibiotic based on vancomycin that is powerfully effective against vancomycin-resistant strains of MRSA and other disease-causing bacteria.

18 Sep 2014

MOVANTIK tablets get FDA approval for treatment of OIC in patients with chronic, non-cancer pain

Nektar Therapeutics reported today that partner AstraZeneca today announced that the US Food and Drug Administration (FDA) approved MOVANTIK (naloxegol) tablets as the first once-daily oral peripherally-acting mu-opioid receptor antagonist (PAMORA) medication for the treatment of opioid-induced constipation (OIC), in adult patients with chronic, non-cancer pain.

16 Sep 2014

Organizations across U.S. participating in Go Gold for Childhood Cancer Awareness campaign

Organizations in Atlanta – and across the country – are participating in the Go Gold for Childhood Cancer Awareness campaign this September. Coordinating this effort is the Aflac Cancer Center of Children's Healthcare of Atlanta along with the Stop Childhood Cancer Alliance that was created to be the driving force in increasing awareness of childhood cancer and enhancing support for the young people fighting this disease, as well as the clinicians and researchers helping them.

2 Sep 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

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