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.

Common pain and anti-inflammation drugs may slow cancer growth

Scientists from the Florida campus of The Scripps Research Institute have found that one of the most widely prescribed pain and anti-inflammation drugs slows the growth rate of a specific kind of cancer in animal models and suggests the medication could have the same effect on other types of tumors.

26 May 2016

Jumping genes may play crucial role in generation of cancer

For more than 50 years, scientists have known of the existence of "jumping genes," strands of DNA material that can move from one location in the genome to another.

11 May 2016

TSRI scientists design potential drug candidate to treat triple negative breast cancer

In a development that could lead to a new generation of drugs to precisely treat a range of diseases, scientists from the Florida campus of The Scripps Research Institute have for the first time designed a drug candidate that decreases the growth of tumor cells in animal models in one of the hardest to treat cancers—triple negative breast cancer.

10 May 2016

TSRI study reveals important traits in LCMV, Lassa virus

For the first time, scientists at The Scripps Research Institute (TSRI) have solved the structure of the biological machinery used by a common virus to recognize and attack human host cells.

27 Apr 2016

Wellderly study finds link between cognitive decline genes and healthy aging

An eight-year-long accrual and analysis of the whole genome sequences of healthy elderly people, or "Wellderly," has revealed a higher-than-normal presence of genetic variants offering protection from cognitive decline, researchers from the Scripps Translational Science Institute (STSI) reported today in the journal Cell.

22 Apr 2016

European Medicines Agency approves first-ever treatment for hereditary factor X deficiency

Bio Products Laboratory, Limited (BPL) today announced that the European Medicines Agency has granted marketing authorisation for Coagadex.

31 Mar 2016

TSRI study shows hollowed-out version of CPMV could be effective in human therapies

Viruses aren't always bad. In fact, scientists can harness the capabilities of some viruses for good—modifying the viruses to carry drug molecules, for example.

29 Mar 2016

Scientists identify two enzymes that appear to play role in metabolism, inflammation

A team led by scientists at The Scripps Research Institute and the Salk Institute for Biological Studies has discovered two enzymes that appear to play a role in metabolism and inflammation—and might someday be targeted with drugs to treat type 2 diabetes and inflammatory disorders.

29 Mar 2016

Engineered HIV vaccine protein may prevent HIV infection

Some people infected with HIV naturally produce antibodies that effectively neutralize many strains of the rapidly mutating virus, and scientists are working to develop a vaccine capable of inducing such "broadly neutralizing" antibodies that can prevent HIV infection.

28 Mar 2016

TSRI scientists develop new process to synthesize naturally occurring compound phorbol

In a landmark feat of chemical synthesis, scientists at The Scripps Research Institute have developed a 19-step process for making the naturally occurring compound phorbol in the laboratory, in quantities that are useful for pharmaceutical research.

24 Mar 2016

Research: X and Y DNA swapping may occur more often than previously thought

It turns out that the rigid "line in the sand" over which the human sex chromosomes---the Y and X--- go to avoid crossing over is a bit blurrier than previously thought. Contrary to the current scientific consensus, Arizona State University assistant professor Melissa Wilson Sayres has led a research team that has shown that X and Y DNA swapping may occur much more often.

24 Mar 2016

UC San Diego Health participates in nationwide clinical study on hemophilia B gene therapy

The Hemophilia and Thrombosis Treatment Center at UC San Diego Health has joined a nationwide clinical trial testing a potential gene therapy that may one day provide a better and long-lasting treatment for people with hemophilia B.

23 Mar 2016

Novel compound shows promise as potential treatment for acute myeloid leukemia

A novel compound has shown promise in preclinical studies as a treatment for acute myeloid leukemia, more than doubling median days of survival even in a drug-resistant form of the disease.

21 Mar 2016

Inherited gene after black plague may help treat HIV patients co-infected with hepatitis C

The Black Death swept Europe in the 14th century eliminating up to half of the population but it left genetic clues that now may aid a University of Cincinnati researcher in treating HIV patients co-infected with hepatitis C using an anti-retroviral drug therapy.

7 Mar 2016

Idelvion approved for use in children and adults with Hemophilia B

The U.S. Food and Drug Administration today approved Idelvion, Coagulation Factor IX (Recombinant), Albumin Fusion Protein, for use in children and adults with Hemophilia B. Idelvion is the first coagulation factor-albumin fusion protein product to be approved, and the second Factor IX fusion protein product approved in the U.S. that is modified to last longer in the blood.

7 Mar 2016

Modified protein appears to reverse liver fibrosis, cirrhosis in rats

A protein modified to increase the amount of time it circulates in the bloodstream appears to reverse liver fibrosis and cirrhosis in rats, according to results of a study led by Johns Hopkins researchers.

3 Mar 2016

Baxalta seeks FDA approval for ADYNOVATE to treat children with hemophilia A and for use in surgical settings

Baxalta Incorporated, a global biopharmaceutical leader dedicated to delivering transformative therapies to patients with orphan diseases and underserved conditions, announced today that it has submitted supplemental Biologics License Applications (sBLAs) to the U.S. Food and Drug Administration seeking approval for the use of ADYNOVATE [Antihemophilic Factor (Recombinant), PEGylated] to treat children under the age of 12 with hemophilia A and for use in surgical settings.

26 Feb 2016

Sangamo presents immunological data from SB-728-T HIV clinical study at CROI 2016

Sangamo BioSciences, Inc., the leader in therapeutic genome editing, announced the presentation of immunological data from the Company's clinical trials of SB-728-T, a ZFP Therapeutic designed to provide functional control of HIV.

25 Feb 2016

New study focuses on safety of using induced pluripotent stem cells in patients

A new study led by scientists at The Scripps Research Institute and the J. Craig Venter Institute shows that the act of creating pluripotent stem cells for clinical use is unlikely to pass on cancer-causing mutations to patients.

22 Feb 2016

TSRI's Courtney Miller to receive Presidential Early Career Award for Scientists and Engineers

Courtney Miller, associate professor on the Florida campus of The Scripps Research Institute, has been selected to receive a Presidential Early Career Award for Scientists and Engineers, the highest honor bestowed by the United States government on young professionals at the outset of their independent research careers.

19 Feb 2016