Hemophilia B News and Research

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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.

18 Jan 2016

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

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

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

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

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

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

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

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

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

Hemophilia B News and Research

TSRI researchers develop new technique for modifying complex drug molecules

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

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

INP achieves 80% survival rate in acute lymphoblastic leukemia patients

Adynovate approved for patients with Hemophilia A

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

Rice scientists solve long-standing mystery about hemophilia protein

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

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

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

Promising drug candidate for chronic intractable itch

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

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

New technology advances genome engineering

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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