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.

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.

12 Jun 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.

30 May 2015

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.

28 May 2015

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.

24 May 2015

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.

8 May 2015

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.

8 May 2015

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)].

5 May 2015

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

29 Apr 2015

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.

29 Apr 2015

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.

28 Apr 2015

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.

From Baxter International Inc. 27 Apr 2015

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).

From Baxter International Inc. 22 Apr 2015

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.

From Baxter International Inc. 16 Apr 2015

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

Hemophilia News and Research

Further Reading

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

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

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

CSL Behring awarded NHF's 2015 Corporate Leadership Award

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

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

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

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

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

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

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

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

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

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

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

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

'Turbocharged' protein holds promise in hemophilia gene therapy

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

TSRI chemists develop innovative method for building new drug molecules

TSRI researchers create picture of whole dynein-dynactin structure

Achieving and maintaining ISO/IEC 27001 and BS 10012 certifications

Expanding research and clinical options for children with cancer

Examining how pain could play a direct protective role in the gut

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