Hemophilia A News and Research

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European Medicines Agency approves first-ever treatment for hereditary factor X deficiency

Bio Products Laboratory, Limited (BPL) today announced that the European Medicines Agency has granted marketing authorisation for Coagadex.

31 Mar 2016

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.

24 Mar 2016

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.

23 Mar 2016

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.

7 Mar 2016

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.

7 Mar 2016

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.

3 Mar 2016

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.

26 Feb 2016

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).

8 Feb 2016

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.

21 Jan 2016

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.

20 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

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

Hemophilia A News and Research

European Medicines Agency approves first-ever treatment for hereditary factor X deficiency

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

UC San Diego Health participates in nationwide clinical study on hemophilia B gene therapy

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

Idelvion approved for use in children and adults with Hemophilia B

Modified protein appears to reverse liver fibrosis, cirrhosis in rats

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

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

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

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

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

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

Expanding research and clinical options for children with cancer

Examining how pain could play a direct protective role in the gut

From Puberty to Menopause: Clue’s CEO, Audrey Tsang on the Power of Femtech

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