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.

Scientists identify brain molecule that triggers schizophrenia-like behaviors, brain changes

Scientists at The Scripps Research Institute have identified a molecule in the brain that triggers schizophrenia-like behaviors, brain changes and global gene expression in an animal model. The research gives scientists new tools for someday preventing or treating psychiatric disorders such as schizophrenia, bipolar disorder and autism.

8 Apr 2015

Novo Nordisk announces U.S. launch of Novoeight for people living with hemophilia A

Novo Nordisk today announced the company will launch Novoeight (Antihemophilic Factor [Recombinant]) in the United States for people living with hemophilia A.

From Novo Nordisk 26 Mar 2015

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.

23 Mar 2015

'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.

13 Mar 2015

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.

12 Mar 2015

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.

12 Mar 2015

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.

12 Mar 2015

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.

11 Mar 2015

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.

From Baxter International Inc. 9 Mar 2015

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.

5 Mar 2015

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.

4 Mar 2015

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.

3 Mar 2015

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.

28 Feb 2015

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.

27 Feb 2015

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.

18 Feb 2015

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.

5 Feb 2015

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.

4 Feb 2015

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.

3 Feb 2015

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.

31 Jan 2015

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.

15 Jan 2015