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.

Novel therapy decreases incidence of bleeding by 87% in hemophilia A patients with inhibitors

An international team of hematologists including Guy Young, MD, of Children's Hospital Los Angeles, has found that in patients with hemophilia A with inhibitors, a novel therapy called emicizumab, decreases incidence of bleeding episodes by 87%.

10 Jul 2017

Abbott introduces Sekisui CP3000 fully automated coagulation system for accurate test results

Today, Abbott announced that the Sekisui CP3000 coagulation system, a fully automated analyzer that tests bleeding and clotting function in blood, is now available in Europe, the Middle East, and Asia Pacific.

23 Jun 2017

International team to set how gene therapies in hemophilia should be measured for effectiveness

An international team involving dozens of stakeholders from patients and policy makers to payers and government agencies are getting together to set how gene therapies in hemophilia should be measured for effectiveness.

20 Jun 2017

TSRI scientist and partners receive $7.2 million grant to create new RNA-based treatments for ALS

Professor Matthew Disney of the Department of Chemistry on the Florida campus of The Scripps Research Institute, together with scientists from Mayo Clinic's Florida campus and Johns Hopkins School of Medicine, has been awarded $7.2 million from the National Institute of Neurological Disorders and Stroke of The National Institutes of Health to create new RNA-based treatments for the most common form of amyotrophic lateral sclerosis (ALS), as well as a type of frontotemporal dementia (FTD).

15 Jun 2017

Altered virus could help more patients to become eligible for human gene therapy trials

For many patients, participating in gene therapy clinical trials isn't an option because their immune system recognizes and fights the helpful virus used for treatment.

13 Jun 2017

TSRI scientists solve cellular mystery that may have implications for fundamental biology

Scientists at The Scripps Research Institute have solved a cellular mystery that may have important implications for fundamental biology and diseases like ALS.

13 Jun 2017

Anti-heroin vaccine developed at TSRI proven effective in primate models

A vaccine developed at The Scripps Research Institute (TSRI) to block the "high" of heroin has proven effective in non-human primates. This is the first vaccine against an opioid to pass this stage of preclinical testing.

6 Jun 2017

TSRI study reveals first-ever structure of Lassa virus protein

Before Ebola virus ever struck West Africa, locals were already on the lookout for a deadly pathogen: Lassa virus. With thousands dying from Lassa every year-;and the potential for the virus to cause even larger outbreaks- researchers are committed to designing a vaccine to stop it.

1 Jun 2017

TSRI professor receives $2 million grant to study neurofibromatosis type I

Assistant Professor Seth Tomchik of the Florida campus of The Scripps Research Institute (TSRI) has received $2 million in funding from the National Institutes of Health's National Institute of Neurological Disorders and Stroke.

31 May 2017

TSRI scientists give new superpowers to lifesaving antibiotic

Scientists at The Scripps Research Institute have given new superpowers to a lifesaving antibiotic called vancomycin, an advance that could eliminate the threat of antibiotic-resistant infections for years to come.

30 May 2017

Scripps scientists make important advance in HIV vaccine design

Scientists at The Scripps Research Institute (TSRI) have made another important advance in HIV vaccine design. The development was possible thanks to previous studies at TSRI showing the structures of a protein on HIV’s surface, called the envelope glycoprotein. The scientists used these structures to design a mimic of the viral protein from a different HIV subtype, subtype C, which is responsible for the majority of infections worldwide.

29 May 2017

Researchers uncover structural details that make eye-targeting virus a better tool for future therapies

In their quest to replicate themselves, viruses have gotten awfully good at tricking human cells into pumping out viral proteins. That's why scientists have been working to use viruses as forces for good: to deliver useful genes to human cells and help patients who lack important proteins or enzymes.

12 May 2017

Sangamo Therapeutics and Pfizer team up for development of Hemophilia A gene therapy

Sangamo Therapeutics, Inc. and Pfizer Inc. today announced an exclusive, global collaboration and license agreement for the development and commercialization of gene therapy programs for Hemophilia A, including SB-525, one of Sangamo's four lead product candidates, which Sangamo expects will enter the clinic this quarter.

11 May 2017

Three TSRI scientists receive awards from Royal Society of Chemistry

Three chemists from The Scripps Research Institute- Dale Boger, Jin-Quan Yu and Phil Baran- have received awards from the Royal Society of Chemistry, a renowned professional organization for chemists based in the United Kingdom, with more than 54,000 members worldwide.

10 May 2017

TSRI scientists reveal important role of genetics in brain circuits

Scientists at The Scripps Research Institute in La Jolla have revealed new clues to the wiring of the brain. A team led by Associate Professor Anton Maximov found that neurons in brain regions that store memory can form networks in the absence of synaptic activity.

19 Apr 2017

Novo Nordisk introduces new web-based portal that helps patients track their hemophilia treatment

Novo Nordisk today announced the availability of HemaGo™ XChange, a web-based portal that allows people with hemophilia to connect their circle of care to their treatment and bleed information, promoting collaboration and discussion regarding the management of their bleeding disorder.

From Novo Nordisk 13 Apr 2017

TSRI study could lead to personalized treatments for alcohol dependence

A new study led by scientists at The Scripps Research Institute could help researchers develop personalized treatments for alcoholism and alcohol use disorder.

12 Apr 2017

TSRI scientists find new way to make cell population resistant to HIV

Scientists at The Scripps Research Institute have found a way to tether HIV-fighting antibodies to immune cells, creating a cell population resistant to the virus.

11 Apr 2017

TSRI professor receives $4.8 million grant to advance potential HIV vaccine research

Professor Michael Farzan, co-chair of the Department of Immunology and Microbiology on the Florida campus of The Scripps Research Institute has received $4.8 million in funding through a 2017 Avant-Garde Award for HIV/AIDS research from the National Institutes of Health's National Institute on Drug Abuse.