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

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. [More]
UC San Diego Health participates in nationwide clinical study on hemophilia B gene therapy

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. [More]
Novel compound shows promise as potential treatment for acute myeloid leukemia

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

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. [More]
Idelvion approved for use in children and adults with Hemophilia B

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. [More]
Modified protein appears to reverse liver fibrosis, cirrhosis in rats

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

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. [More]
Sangamo presents immunological data from SB-728-T HIV clinical study at CROI 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. [More]
New study focuses on safety of using induced pluripotent stem cells in patients

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. [More]
TSRI's Courtney Miller to receive Presidential Early Career Award for Scientists and Engineers

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. [More]
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]
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