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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.
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 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]
Merrimack completes submission of MM-398 NDA for treatment of post-gemcitabine metastatic pancreatic cancer

Merrimack completes submission of MM-398 NDA for treatment of post-gemcitabine metastatic pancreatic cancer

Merrimack Pharmaceuticals, Inc. and Baxter International Inc. today jointly announced that Merrimack has completed the rolling submission of the New Drug Application (NDA) for MM-398 (irinotecan liposome injection), also known as "nal-IRI," to the U.S. Food and Drug Administration. [More]
Data from pacritinib Phase 3 PERSIST-1 trial in patients with myelofibrosis to be highlighted at ASCO

Data from pacritinib Phase 3 PERSIST-1 trial in patients with myelofibrosis to be highlighted at ASCO

CTI BioPharma Corp. and Baxter International Inc. today announced that data from the randomized Phase 3 PERSIST-1 trial evaluating the investigational agent pacritinib in patients with myelofibrosis will be highlighted in a late-breaking oral presentation at the upcoming American Society of Clinical Oncology 2015 Meeting (May 29-June 2, 2015 in Chicago, Ill). [More]
Baxter submits BAX 855 NDA to Japan's MHLW for treatment of hemophilia A patients

Baxter submits BAX 855 NDA to Japan's MHLW for treatment of hemophilia A patients

Nektar Therapeutics reported today that partner Baxter International Inc. announced that the company has submitted a new drug application (NDA) to Japan's Ministry of Health, Labour and Welfare for the approval of BAX 855, an investigational, extended half-life recombinant factor VIII (rFVIII) treatment based on ADVATE [Antihemophilic Factor (Recombinant)] for patients over 12 years of age with hemophilia A. [More]
Scientists identify brain molecule that triggers schizophrenia-like behaviors, brain changes

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

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. [More]
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]
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