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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.
Animal study shows link between oxygen-sensing neurons and fat-burning circuit

Animal study shows link between oxygen-sensing neurons and fat-burning circuit

A new study in animal models, led by scientists at The Scripps Research Institute (TSRI), is the first to show that oxygen sensing in the brain has a role in metabolism and sensing an organism's internal state. [More]
FDA clears Sangamo BioSciences' SB-318 IND application for treatment of MPS I

FDA clears Sangamo BioSciences' SB-318 IND application for treatment of MPS I

Sangamo BioSciences, Inc., the leader in therapeutic genome editing, announced that the U.S. Food and Drug Administration has cleared the Company's Investigational New Drug (IND) application for SB-318, a single treatment strategy intended to provide a life-long therapy for Mucopolysaccharidosis Type I (MPS I). [More]
Remicade co-developer funds new microscopy facility on Scripps Florida campus

Remicade co-developer funds new microscopy facility on Scripps Florida campus

The co-developer of Remicade, one of the three top-selling drugs in the world, has donated more than $500,000 to fund what will be known as the Iris and Junming Le Foundation Super-Resolution Microscopy Facility on the Florida campus of The Scripps Research Institute. [More]
TSRI scientists reveal workings of key 'relief-valve' in cells

TSRI scientists reveal workings of key 'relief-valve' in cells

A team led by scientists at The Scripps Research Institute has solved a long-standing mystery in cell biology by showing essentially how a key "relief-valve" in cells does its job. [More]
Gene therapy holds considerable potential for safe, effective treatment of people with factor VII deficiency

Gene therapy holds considerable potential for safe, effective treatment of people with factor VII deficiency

Hematology researchers have used a single injection of gene therapy to correct a rare bleeding disorder, factor VII deficiency, in dogs. This success in large animals holds considerable potential for a safe, effective and long-lasting new treatment in humans with the same bleeding disorder. [More]
Novel gene therapy treatment proves safe, effective for factor VII deficiency

Novel gene therapy treatment proves safe, effective for factor VII deficiency

A single injection. That's all someone with a factor VII deficiency would need for a life-long cure, thanks to a new gene therapy treatment developed in a collaboration of researchers at the University of North Carolina and The Children's Hospital of Philadelphia. [More]
TSRI researchers develop new technique for modifying complex drug molecules

TSRI researchers develop new technique for modifying complex drug molecules

Chemists at The Scripps Research Institute have developed a versatile new technique for making modifications—especially one type of extremely difficult, but much-sought-after modification—to complex drug molecules. [More]
U-M research could lead to new ways of fighting X-linked diseases in girls and women

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. [More]

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. [More]
New $8.5M research grant aims to address patients affected by severe hemophilia A

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. [More]
Women suffering from blood clots can safely take hormone replacement therapy with anticoagulants

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. [More]
Baxalta announces initial results from ADYNOVATE Phase 3 trial for treatment of hemophilia patients

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)]. [More]
CSL submits rIX-FP new drug application to Japan's Pharmaceuticals and Medical Devices Agency

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). [More]
FDA updates blood donor deferral recommendations to help ensure continued safety of blood supply

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. [More]
New study examines single 'transformer' proteins involved in cancer

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

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. [More]
Mutant protein responsible for cystic fibrosis talks to wrong cellular neighbors

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. [More]
TSRI researchers awarded $1.6 million grant to advance preclinical studies of potential heroin vaccine

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. [More]
INP achieves 80% survival rate in acute lymphoblastic leukemia patients

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. [More]
TSRI scientists discover first-in-class compounds that target persistent tuberculosis

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. [More]
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