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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.
Male genetic diversity declined due to wealth, power rather than `survival of fittest`

Male genetic diversity declined due to wealth, power rather than `survival of fittest`

The DNA you inherit from your parents contributes to the physical make-up of your body -- whether you have blue eyes or brown, black hair or red, or are male or female. [More]
'Turbocharged' protein holds promise in hemophilia gene therapy

'Turbocharged' protein holds promise in hemophilia gene therapy

Using gene therapy to produce a mutant human protein with unusually high blood-clotting power, scientists have successfully treated dogs with the bleeding disorder hemophilia, without triggering an unwanted immune response. [More]
Study: New gene therapy safe, effective for patients with hemophilia B

Study: New gene therapy safe, effective for patients with hemophilia B

A multi-year, ongoing study suggests that a new kind of gene therapy for hemophilia B could be safe and effective for human patients. Published in the journal Science Translational Medicine, the research showed that a reprogrammed retrovirus could successfully transfer new factor IX (clotting) genes into animals with hemophilia B to dramatically decrease spontaneous bleeding. [More]
TSRI chemists develop innovative method for building new drug molecules

TSRI chemists develop innovative method for building new drug molecules

Chemists at The Scripps Research Institute have developed a broadly useful technique for building new drug molecules and other chemical products. [More]
TSRI researchers create picture of whole dynein-dynactin structure

TSRI researchers create picture of whole dynein-dynactin structure

A team led by scientists at The Scripps Research Institute has determined the basic structural organization of a molecular motor that hauls cargoes and performs other critical functions within cells. [More]
New injectable polymer could strengthen blood clots

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. [More]
Pacritinib for myelofibrosis meets primary endpoint in Phase 3 PERSIST-1 trial

Pacritinib for myelofibrosis meets primary endpoint in Phase 3 PERSIST-1 trial

CTI BioPharma Corp. and Baxter International Inc. today announced positive top-line results for the primary endpoint from PERSIST-1, the randomized, controlled Phase 3 registration clinical trial examining pacritinib, a next generation oral JAK2/FLT3 multikinase inhibitor, for the treatment of patients with primary or secondary myelofibrosis. [More]
Finding may help advance experimental approach to improving public health

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. [More]
Bayer announces FDA acceptance of BAY 81-8973 BLA for treatment of hemophilia A

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. [More]
TSRI researchers show how ABC transporters cause multidrug resistance

TSRI researchers show how ABC transporters cause multidrug resistance

Cancer patients fear the possibility that one day their cells might start rendering many different chemotherapy regimens ineffective. This phenomenon, called multidrug resistance, leads to tumors that defy treatment. [More]
CSL Behring donates protein therapies to World Federation of Hemophilia

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. [More]
TSRI scientists show how to target weak spots of Marburg virus with future treatments

TSRI scientists show how to target weak spots of Marburg virus with future treatments

Marburg virus is Ebola's deadly cousin. The virus is up to 90 percent lethal—and doctors are desperate for tools to fight it. [More]
TSRI scientists develop new drug candidate against HIV

TSRI scientists develop new drug candidate against HIV

In a remarkable new advance against the virus that causes AIDS, scientists from the Jupiter, Florida campus of The Scripps Research Institute have announced the creation of a novel drug candidate that is so potent and universally effective, it might work as part of an unconventional vaccine. [More]
Alzheimer's brains commonly have many neurons with more DNA and genomic copies of APP gene

Alzheimer's brains commonly have many neurons with more DNA and genomic copies of APP gene

Scientists at The Scripps Research Institute have found diverse genomic changes in single neurons from the brains of Alzheimer's patients, pointing to an unexpected factor that may underpin the most common form of the disease. [More]
FDA accepts Sangamo BioSciences' IND for SB-BCLmR-HSPC genome editing approach

FDA accepts Sangamo BioSciences' IND for SB-BCLmR-HSPC genome editing approach

Sangamo BioSciences, Inc. announced today that an Investigational New Drug (IND) application for the company's SB-BCLmR-HSPC genome editing approach, which is designed to provide a one-time lasting therapy for beta-thalassemia, has been accepted by the U.S. Food and Drug Administration and is now active. [More]
New study helps explain how booster shots trigger immune 'memory' to improve

New study helps explain how booster shots trigger immune 'memory' to improve

The last time you were in the doctor's office for a vaccine booster shot, did you wonder why you needed one? Exactly how booster shots offer long-term protection from viruses has long been a mystery to scientists. [More]
TSRI researchers awarded grant to develop novel method for detecting DNA damage in stem cells

TSRI researchers awarded grant to develop novel method for detecting DNA damage in stem cells

A team at The Scripps Research Institute has won a $1,784,000 grant from the California Institute for Regenerative Medicine (CIRM). The funding will support the development of a new method for detecting DNA damage in stem cells to ensure that only the highest quality cells are used in transplantation or therapy. [More]
TSRI scientists identify novel synthetic compound that reduces activity of a cancer-related protein

TSRI scientists identify novel synthetic compound that reduces activity of a cancer-related protein

Scientists from the Florida campus of The Scripps Research Institute have identified a novel synthetic compound that sharply inhibits the activity of a protein that plays an important role in in the progression of breast and pancreatic cancers. [More]
Biogen Idec, Columbia University Medical Center form $30 million genomics research alliance

Biogen Idec, Columbia University Medical Center form $30 million genomics research alliance

Biogen Idec and Columbia University Medical Center have formed a $30 million strategic alliance to conduct genetics discovery research on the underlying causes of disease and to identify new treatment approaches. As part of this agreement, a sequencing and analysis facility and shared postdoctoral program will be established at Columbia to support collaborative genetics studies. [More]
Janssen, Gilead to jointly develop darunavir-based, single-tablet regimen for HIV treatment

Janssen, Gilead to jointly develop darunavir-based, single-tablet regimen for HIV treatment

Janssen R&D Ireland announced today an amendment to its existing agreement with Gilead Sciences, Inc., initially established in 2011, for the development of a once daily, darunavir-based, single-tablet regimen (STR) for the treatment of people living with HIV. [More]
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