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.

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

New genetic discovery could lead to better treatment for X-linked diseases

Think back to middle school biology class, when you learned that boys have an X and a Y chromosome inside each cell, and girls have two X's.

20 Oct 2015

Interim data from long-term extension Tecfidera® (dimethyl fumarate) study

Biogen (NASDAQ: BIIB) will, this week, present data at the 31st Congress of the European Committee for Treatment and Research in Multiple Sclerosis (ECTRIMS) in Barcelona, Spain, demonstrating the efficacy and safety of TECFIDERA® (dimethyl fumarate) across a broad range of people with relapsing-remitting multiple sclerosis (RRMS).

7 Oct 2015

Promising drug candidate for chronic intractable itch

If you have an itch, you have to scratch it. But that’s a problem for people with a condition called “chronic intractable itch,” where that itchy sensation never goes away—a difficult-to-treat condition closely associated with dialysis and renal failure.

28 Sep 2015

Novo Nordisk announces FDA approval of Tresiba (insulin degludec injection) for diabetes treatment

Novo Nordisk, a world leader in diabetes care, today announced that the U.S. Food and Drug Administration approved the new drug application for Tresiba (insulin degludec injection), a once-daily, long-acting basal insulin. Tresiba is indicated for use alone, or in combination with oral antidiabetic medicines or bolus insulin, and is approved for glycemic control in adults with type 1 and type 2 diabetes. Tresiba provides a long duration of action beyond 42 hours.

From Novo Nordisk 26 Sep 2015

Shire and Sangamo revise agreement to expedite development of ZFP Therapeutics for Huntington's disease, hemophilia A and B

Sangamo BioSciences, Inc., a leader in therapeutic genome editing, announced today that the company and its collaborator, Shire plc, have agreed to revise their January 2012 collaboration and license agreement to expedite the development of ZFP Therapeutics for hemophilia A and B and Huntington's disease.

2 Sep 2015

Nektar receives $40 million cash payment from first commercial sale of MOVENTIG (naloxegol)

Nektar Therapeutics announced today that it has received a $40 million cash payment under a license agreement with AstraZeneca. The payment was triggered by the first commercial sale of MOVENTIG (naloxegol) in Germany.

1 Sep 2015

First patient enrolled in CSL Behring's rVIIa-FP Phase II/III study to treat patients with hemophilia A or B with inhibitors

CSL Behring announced today that the first patient has been enrolled in its Phase II/III clinical study evaluating the pharmacokinetics (PK), efficacy, and safety of the company's recombinant fusion protein linking coagulation factor VIIa with albumin (rVIIa-FP) for on-demand treatment in patients with congenital hemophilia A or B who have developed an inhibitor to factor VIII or factor IX replacement therapy.

26 Aug 2015

TSRI, Janssen collaborate to find universal flu vaccine

Scientists from The Scripps Research Institute and the Janssen Pharmaceutical Companies of Johnson & Johnson have found a way to induce antibodies to fight a wide range of influenza subtypes—work that could one day eliminate the need for repeated seasonal flu shots.

24 Aug 2015

Improved method for building proteins into antibodies

Some proteins exist so fleetingly in the bloodstream that they can't be given effectively as therapies. However, building them into larger proteins, such as antibodies, can make them persist long enough to be useful. Now a team led by scientists at The Scripps Research Institute (TSRI) has devised an improved method for accomplishing this protein-engineering feat.

14 Aug 2015

New technology advances genome engineering

In his mind, Basil Hubbard can already picture a new world of therapeutic treatments for millions of patients just over the horizon. It's a future in which diseases like muscular dystrophy, cystic fibrosis and many others are treated permanently through the science of genome engineering.

11 Aug 2015

New analysis examines pharmacy expenditures for publicly insured children with serious chronic illness

In an analysis of expenditures for outpatient pharmacy products used by publicly insured children with serious chronic illness in California, treating hemophilia accounted for about 40 percent of expenditures but included just 0.4 percent of the group studied, suggesting a need to improve pricing for this and other effective yet high-cost medications, according to a study in the July 28 issue of JAMA.

29 Jul 2015

TSRI scientists awarded grant to explore therapeutic potential of protein receptors in Parkinson's disease, other disorders

Scientists from the Florida campus of The Scripps Research Institute have been awarded nearly $1.5 million from the National Institute of General Medical Sciences of the National Institutes of Health to explore the therapeutic potential of a class of proteins that play essential roles in the regulation and maintenance of human health.

28 Jul 2015

Researchers find way to reverse clotting factor deficiency that triggers hemophilia A

Sufferers of hemophilia live in a perpetual state of stress and anxiety: their joints wear down prematurely and they have bleeding episodes that feel like they will never end. Their bodies lack the ability to make the clotting factor responsible for the coagulation of blood so any cut or bruise can turn into an emergency without immediate treatment.

24 Jul 2015