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.

U-M research could lead to new ways of fighting X-linked diseases in girls and women

Nearly every girl and woman on Earth carries two X chromosomes in nearly every one of her cells -- but one of them does (mostly) nothing. That's because it's been silenced, keeping most of its DNA locked up and unread like a book in a cage.

11 Jan 2016

Several therapies for hemophilia A, hemophilia B and hemophilia with inhibitors to be launched between 2015-2025

Decision Resources Group forecasts that several therapies will launch for hemophilia A, hemophilia B and hemophilia with inhibitors in the United States and Europe during our 2015-2025 study period.

29 Dec 2015

New $8.5M research grant aims to address patients affected by severe hemophilia A

People with hemophilia have prolonged abnormal bleeding as a result of trauma. Hemophilia A, also called factor VIII (FVIII) deficiency, is the most common form of the genetic disorder caused by missing or defective blood clotting protein called factor VIII.

23 Dec 2015

Women suffering from blood clots can safely take hormone replacement therapy with anticoagulants

New research published online today in Blood, the Journal of the American Society of Hematology (ASH), is the first to demonstrate that women on blood thinners can take estrogen-containing contraception or hormone replacement therapy without an increased risk of blood clots or uterine bleeding.

23 Dec 2015

Baxalta announces initial results from ADYNOVATE Phase 3 trial for treatment of hemophilia patients

Baxalta Incorporated, a global biopharmaceutical leader dedicated to delivering transformative therapies to patients with orphan diseases and underserved conditions, today announced initial results from a Phase 3 clinical trial of ADYNOVATE [Antihemophilic Factor (Recombinant), PEGylated], an extended circulating half-life recombinant Factor VIII (rFVIII) treatment for hemophilia A based on ADVATE [Antihemophilic Factor (Recombinant)].

22 Dec 2015

CSL submits rIX-FP new drug application to Japan's Pharmaceuticals and Medical Devices Agency

Global biotherapeutics leader CSL Behring announced today that the company has submitted its new drug application to Japan's Pharmaceuticals and Medical Devices Agency for its investigational fusion protein linking recombinant coagulation factor IX with recombinant albumin (rIX-FP).

22 Dec 2015

FDA updates blood donor deferral recommendations to help ensure continued safety of blood supply

Today, the U.S. Food and Drug Administration issued final guidance outlining updated blood donor deferral recommendations to reflect the most current scientific evidence and to help ensure continued safety of the blood supply by reducing the risk of human immunodeficiency virus (HIV) transmission by blood and blood products.

22 Dec 2015

New study examines single 'transformer' proteins involved in cancer

A new study led by scientists at The Scripps Research Institute and St. Jude Children's Research Hospital shows how a protein involved in cancer twists and morphs into different structures.

22 Dec 2015

TSRI-led study shows how different drugs regulate protein linked to asthma, obesity and diabetes

A new study, led by scientists at The Scripps Research Institute, shows how different pharmaceutical drugs hit either the "on" or "off" switch of a signaling protein linked to asthma, obesity and type 2 diabetes.

11 Dec 2015

Mutant protein responsible for cystic fibrosis talks to wrong cellular neighbors

Scientists at The Scripps Research Institute have found evidence that a mutant protein responsible for most cases of cystic fibrosis is so busy "talking" to the wrong cellular neighbors that it cannot function normally and is prematurely degraded.

1 Dec 2015

TSRI researchers awarded $1.6 million grant to advance preclinical studies of potential heroin vaccine

Now researchers at The Scripps Research Institute have been awarded a prestigious Translational Avant-Garde Award, which supports the development of medications for substance abuse disorders, from the National Institutes of Health National Institute on Drug Abuse.

20 Nov 2015

INP achieves 80% survival rate in acute lymphoblastic leukemia patients

The National Institute of Pediatrics in Mexico, has achieved 80 percent survival rate in patients with acute lymphoblastic leukemia, a major oncological illness that affects children.

19 Nov 2015

TSRI scientists discover first-in-class compounds that target persistent tuberculosis

Tuberculosis has been infecting humans for several millennia, making it one of the most horribly successful diseases in history. Today, it is still a major killer, responsible for some 1.5 million deaths each year.

18 Nov 2015

Adynovate approved for patients with Hemophilia A

The U.S. Food and Drug Administration today approved Adynovate, Antihemophilic Factor (Recombinant), PEGylated for use in adults and adolescents, aged 12 years and older, who have Hemophilia A. Adynovate is modified to last longer in the blood and potentially require less frequent injections than unmodified Antihemophilic Factor when used to reduce the frequency of bleeding.

15 Nov 2015

St. Jude and Scripps Research Institute scientists help launch Human Dark Proteome Initiative

Scientists at St. Jude Children’s Research Hospital, The Scripps Research Institute (TSRI) and other institutions today announced the launch of the Human Dark Proteome Initiative (HDPI). The initiative aims to accelerate research into biology’s “invisible mass” to provide novel insights into cell function and a new frontier in drug discovery.

9 Nov 2015

Scientists show how specialized cells help each other survive under stress

A team led by scientists from the Florida campus of The Scripps Research Institute and the University of Pittsburgh has shown for the first time how one set of specialized cells survives under stress by manipulating the behavior of key immune system cells.

4 Nov 2015

TSRI scientists awarded NIH grant to investigate molecular machinery involved in cancer metastasis

Cancer metastasis occurs when tumor cells escape from a primary tumor, via the blood stream, and establish tumors in secondary locations. When the metastases are found in distant tissues and organs, doctors call this stage IV cancer, and the survival rate is low.

3 Nov 2015

Rice scientists solve long-standing mystery about hemophilia protein

Rice University scientists have solved a long-standing mystery about where the body stores and deploys blood-clotting factor VIII, a protein that about 80 percent of hemophiliacs cannot produce due to genetic defects.

3 Nov 2015

New study from TSRI and Salk points to cause of debilitating nerve disease

Scientists at The Scripps Research Institute (TSRI) and the Salk Institute for Biological Studies have discovered how a mutant protein triggers nerve damage in a subtype of Charcot-Marie-Tooth (CMT) diseases, a group of currently untreatable conditions that cause loss of function in a person’s hands and feet.

22 Oct 2015

TSRI researchers find way to change leukemia cells into leukemia-killing immune cells

Scientists at The Scripps Research Institute have found a way to change leukemia cells into leukemia-killing immune cells. The surprise finding could lead to a powerful new therapy for leukemia and possibly other cancers.

20 Oct 2015

Hemophilia News and Research

Further Reading

U-M research could lead to new ways of fighting X-linked diseases in girls and women

Several therapies for hemophilia A, hemophilia B and hemophilia with inhibitors to be launched between 2015-2025

New $8.5M research grant aims to address patients affected by severe hemophilia A

Women suffering from blood clots can safely take hormone replacement therapy with anticoagulants

Baxalta announces initial results from ADYNOVATE Phase 3 trial for treatment of hemophilia patients

CSL submits rIX-FP new drug application to Japan's Pharmaceuticals and Medical Devices Agency

FDA updates blood donor deferral recommendations to help ensure continued safety of blood supply

New study examines single 'transformer' proteins involved in cancer

TSRI-led study shows how different drugs regulate protein linked to asthma, obesity and diabetes

Mutant protein responsible for cystic fibrosis talks to wrong cellular neighbors

TSRI researchers awarded $1.6 million grant to advance preclinical studies of potential heroin vaccine

INP achieves 80% survival rate in acute lymphoblastic leukemia patients

TSRI scientists discover first-in-class compounds that target persistent tuberculosis

Adynovate approved for patients with Hemophilia A

St. Jude and Scripps Research Institute scientists help launch Human Dark Proteome Initiative

Scientists show how specialized cells help each other survive under stress

TSRI scientists awarded NIH grant to investigate molecular machinery involved in cancer metastasis

Rice scientists solve long-standing mystery about hemophilia protein

New study from TSRI and Salk points to cause of debilitating nerve disease

TSRI researchers find way to change leukemia cells into leukemia-killing immune cells

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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