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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.
New analysis examines pharmacy expenditures for publicly insured children with serious chronic illness

New analysis examines pharmacy expenditures for publicly insured children with serious chronic illness

In an analysis of expenditures for outpatient pharmacy products used by publicly insured children with serious chronic illness in California, treating hemophilia accounted for about 40 percent of expenditures but included just 0.4 percent of the group studied, suggesting a need to improve pricing for this and other effective yet high-cost medications, according to a study in the July 28 issue of JAMA. [More]
TSRI scientists awarded grant to explore therapeutic potential of protein receptors in Parkinson's disease, other disorders

TSRI scientists awarded grant to explore therapeutic potential of protein receptors in Parkinson's disease, other disorders

Scientists from the Florida campus of The Scripps Research Institute have been awarded nearly $1.5 million from the National Institute of General Medical Sciences of the National Institutes of Health to explore the therapeutic potential of a class of proteins that play essential roles in the regulation and maintenance of human health. [More]
Researchers find way to reverse clotting factor deficiency that triggers hemophilia A

Researchers find way to reverse clotting factor deficiency that triggers hemophilia A

Sufferers of hemophilia live in a perpetual state of stress and anxiety: their joints wear down prematurely and they have bleeding episodes that feel like they will never end. Their bodies lack the ability to make the clotting factor responsible for the coagulation of blood so any cut or bruise can turn into an emergency without immediate treatment. [More]
TSRI awarded two grants to support development of effective vaccine against HIV/AIDS

TSRI awarded two grants to support development of effective vaccine against HIV/AIDS

The Scripps Research Institute has been awarded two grants from the Bill & Melinda Gates Foundation totaling more than $4.5 million to fund efforts to develop a vaccine against HIV/AIDS. [More]
TSRI scientists awarded two grants to develop new therapeutic target to reduce latent HIV infection

TSRI scientists awarded two grants to develop new therapeutic target to reduce latent HIV infection

Scientists from the Florida campus of The Scripps Research Institute have been awarded a pair of grants totaling nearly $2.8 million from the National Institute of Allergy and Infectious Diseases of The National Institutes of Health to develop a new therapeutic agent to reduce latent levels of HIV that hide from the immune system in infected individuals. [More]
Novo Nordisk presents new data detailing the impact of hemophilia on patients, caregivers

Novo Nordisk presents new data detailing the impact of hemophilia on patients, caregivers

Today, Novo Nordisk presented new data detailing the impact hemophilia has on both caregivers and those living with the disease. [More]
Scripps Florida scientists receive $3.5 million to accelerate development of anti-diabetic compounds

Scripps Florida scientists receive $3.5 million to accelerate development of anti-diabetic compounds

Scientists from the Florida campus of The Scripps Research Institute (TSRI) have been awarded $3.5 million from the National Institute of Diabetes and Digestive and Kidney Diseases of the National Institutes of Health to accelerate development of a new class of anti-diabetic compounds. [More]
CSL Behring presents Phase I/III rVIII-SingleChain hemophilia A data at 2015 ISTH Congress

CSL Behring presents Phase I/III rVIII-SingleChain hemophilia A data at 2015 ISTH Congress

CSL Behring today presented data from a Phase I/III study on the efficacy and safety of its novel investigational recombinant factor VIII single chain in adolescents and adults with hemophilia A during a late breaking abstract session at the 2015 International Society on Thrombosis and Haemostasis (ISTH) Congress. [More]
Alnylam presents positive Phase 1 ALN-AT3 trial results in hemophilia at ISTH 2015 Congress

Alnylam presents positive Phase 1 ALN-AT3 trial results in hemophilia at ISTH 2015 Congress

Additional study results from 12 hemophilia A and B subjects demonstrated that subcutaneous administration of ALN-AT3 achieved potent and dose-dependent knockdown of AT of up to 86%. AT knockdown was highly durable, with effects lasting over two months after the last dose, supporting further evaluation of a once-monthly subcutaneous dose regimen. [More]
Experimental AIDS vaccine candidate stimulates immune system to block infection in animal model

Experimental AIDS vaccine candidate stimulates immune system to block infection in animal model

New research led by scientists at The Scripps Research Institute, International AIDS Vaccine Initiative and The Rockefeller University shows that an experimental vaccine candidate can stimulate the immune system to block HIV infection in mice. [More]
New TSRI study integrates neuroscience and psychological research to understand sleep, memory

New TSRI study integrates neuroscience and psychological research to understand sleep, memory

In Macbeth, Shakespeare describes sleep as "the death of each day's life," but he may have gotten it wrong. Sleep, as it turns out, may be the one thing that keeps our memories alive and intact. [More]
Positive results from pacritinib Phase 3 trial for treatment of myelofibrosis presented at ASCO 2015

Positive results from pacritinib Phase 3 trial for treatment of myelofibrosis presented at ASCO 2015

CTI BioPharma Corp. and Baxter International's Bioscience business today announced data from PERSIST-1 – a randomized Phase 3 registration-directed trial examining pacritinib for the treatment of myelofibrosis – in a late-breaking oral session at the 51st Annual Meeting of the American Society of Clinical Oncology, May 29-June 2, 2015 in Chicago, Ill. [More]
TSRI scientists awarded $2.2 million NIH grant to advance innovative approach to obesity treatment

TSRI scientists awarded $2.2 million NIH grant to advance innovative approach to obesity treatment

Scientists from the Florida campus of The Scripps Research Institute have been awarded nearly $2.2 million by the National Institute of Diabetes and Digestive and Kidney Diseases of the National Institutes of Health to advance an innovative approach to the treatment of obesity, a serious health problem that affects more than one-third of all Americans. [More]
CSL Behring awarded NHF's 2015 Corporate Leadership Award

CSL Behring awarded NHF's 2015 Corporate Leadership Award

The National Hemophilia Foundation has awarded CSL Behring its 2015 Corporate Leadership Award as recognition for the company's longstanding and unwavering commitment to advancing science and improving the care of the bleeding disorders community. The award was accepted by Paul Perreault, CEO and Managing Director, CSL Limited, during the NHF Annual Spring Soiree in New York City on May 21. [More]
Overexpression of cyclin E protein could lead to breast cancer, leukemia

Overexpression of cyclin E protein could lead to breast cancer, leukemia

A new study led by scientists at The Scripps Research Institute sheds light on the cause of some cancers, including breast cancer and leukemia. [More]
Removing bacterial biofilms could help prevent and treat colon cancers, study suggests

Removing bacterial biofilms could help prevent and treat colon cancers, study suggests

A team led by scientists at The Scripps Research Institute and Johns Hopkins University School of Medicine has uncovered a big clue to how bacteria may promote some colon cancers. [More]

CMC Biologics to supply bulk drug substance for Emergent BioSolutions' IXINITY

CMC Biologics, Inc., a global CMO dedicated to process development and production of biopharmaceuticals for clinical and commercial use, today announced that it will supply bulk drug substance for Emergent BioSolutions' recently FDA-approved product, IXINITY® [coagulation factor IX (recombinant)]. [More]
Sangamo BioSciences to present data on ZFP Therapeutic platform at ASGCT meeting

Sangamo BioSciences to present data on ZFP Therapeutic platform at ASGCT meeting

Sangamo BioSciences, Inc. announced that data from clinical, preclinical and research-stage programs focused on the development of ZFP Therapeutics will be presented at the 18th Annual Meeting of the American Society of Gene and Cell Therapy [More]
New TSRI study points to promising new therapeutic target for Alzheimer's disease

New TSRI study points to promising new therapeutic target for Alzheimer's disease

Taking a new approach, scientists from the Florida campus of The Scripps Research Institute (TSRI) have uncovered some surprising details of a group of compounds that have shown significant potential in stimulating the growth of brain cells and memory restoration in animal models that mimic Alzheimer's disease. [More]
TSRI study provides new insight into preventing diseases that cause vision loss in adults

TSRI study provides new insight into preventing diseases that cause vision loss in adults

A new study from scientists at The Scripps Research Institute shows that nerve cells and blood vessels in the eye constantly "talk" to each other to maintain healthy blood flow and prevent disease. [More]
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