Hemophilia B News and Research

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New BlooDe device helps study plugging capacity of platelets

Various diseases can cause hemorrhages or thromboses, sometimes fatal, resulting in particular from complications during surgery.

21 Jun 2019

FDA authorizes marketing of first medical device for pain relief in adolescents with IBS

The U.S. Food and Drug Administration today permitted marketing of the first medical device to aid in the reduction of functional abdominal pain in patients 11-18 years of age with irritable bowel syndrome when combined with other therapies for IBS.

10 Jun 2019

Coagulation factor VIIa prevents bleeds in hemophilia animal models

Hematology researchers have further refined how a treatment currently used on an urgent basis to control bleeding in hemophilia patients holds promise as a preventive treatment as well.

16 Apr 2019

Liver tissue model can help investigate the effects of RNA interference

Novel therapies based on a process known as RNA interference hold great promise for treating a variety of diseases by blocking specific genes in a patient's cells. Many of the earliest RNAi treatments have focused on diseases of the liver, because RNA-carrying particles tend to accumulate in that organ.

6 Mar 2019

Researchers are developing and testing novel gene therapy to cure Crigler-Najjar syndrome

Crigler-Najjar syndrome affects about one in a million people at birth. Because of a defective gene, these individuals lack an essential enzyme in their liver to eliminate bilirubin, a toxic bile pigment that accumulates in all body tissues causing jaundice and potentially leading to irreversible and lethal neurological damage.

28 Feb 2019

Genetic relic of the 'Black Death' may provide clues for treating fatal liver scarring

A gene mutation that is believed to have safeguarded some people in 14th century Europe from the bubonic plague today may be protecting HIV patients co-infected with hepatitis C from potentially fatal liver scarring, says a University of Cincinnati College of Medicine physician-scientist.

3 Dec 2018

New clinical practice guidelines for managing venous thromboembolism released

Venous thromboembolism, a term referring to blood clots in the veins, is a highly prevalent and far-reaching public health problem that can cause disability and death.

28 Nov 2018

FDA approves HEMLIBRA for treatment of hemophilia A

Chugai Pharmaceutical Co., Ltd. announced today that the U.S. Food and Drug Administration (FDA) has approved HEMLIBRA (US generic name: emicizumab-kxwh), a treatment for hemophilia A created by Chugai, for routine prophylaxis to prevent or reduce the frequency of bleeding episodes in adults and children, ages newborn and older, with hemophilia A without factor VIII inhibitors, administered once weekly, every two weeks, or every four weeks.

5 Oct 2018

Hopkins gastroenterologists use endoscopic procedure to deliver therapeutic genes to the liver

Fixing or replacing faulty genes has emerged as a key to unlocking cures for numerous devastating diseases. But if the new, engineered genes can't find their way into the patient's genomic sequence, they won't help.

3 Oct 2018

Novel method using big data tools shows promising routes for research

A group of researchers affiliated to the University of São Paulo - campus Ribeirão Preto, Brazil, used big data tools such as data mining and network analysis to develop a method to help scientists and companies identify technological routes, trends and partnerships in any knowledge area. For this purpose, it collected information from patent databases around the world.

3 Sep 2018

Subcutaneous injection of new protein helps treat hemophilia more effectively

Patients with hemophilia bleed spontaneously or following trauma. The most common sites of bleeding are into joints, but bleeds can also happen into any tissue in the body including the muscles, brain, or eyes.

31 Aug 2018

Promising data supports development of AAVhu37-based gene therapy for hemophilia A

Gene therapy using an optimized adeno-associated virus to deliver the human factor VIII gene to cynomolgus macaques showed a substantial increase in hFVIII expression and no detectable antibody response for 30 weeks in some animals.

21 Aug 2018

RCSI and Bayer collaborate to improve treatments for people with hemophilia

RCSI and Bayer have today announced a research collaboration that aims to improve treatments for people with severe haemophilia.

9 Aug 2018

Researchers use genome editing to reduce cholesterol levels in animal model

Using genome editing to inactivate a protein called PCSK9 effectively reduces cholesterol levels in rhesus macaques, a species of monkey, according to researchers from the Perelman School of Medicine at the University of Pennsylvania.

10 Jul 2018

ABPI associated with NHS for 70 years in pharmaceutical research

In this special year, both the NHS and the Association of the British Pharmaceutical Industry are celebrating our respective 70th anniversaries. The histories of these two organizations have been inextricably linked since 1948 and our relationship with the NHS continues to go from strength to strength.

25 Jun 2018

LogicBio team to present data on preclinical effectiveness of GeneRide platform

LogicBio Therapeutics, Inc., a genetic medicine company founded to develop safe medicines with lasting therapeutic benefit for children with life-threatening diseases, today announced it will be presenting data on the company's technology and robust preclinical pipeline at the annual meeting of the American Society of Gene & Cell Therapy at the Hilton Chicago, from May 16-19, 2018.

14 May 2018

Breakthrough study promises to bring gene-editing technology much closer to therapeutic reality

Imagine a future where a guided biomachine put into your body seeks out defective gene sequences in each cell and edits in the correct information with precision accuracy.

13 Apr 2018

UCLA-developed nanospears could make gene therapies safer, faster and more cost-effective

UCLA scientists have developed a new method that utilizes microscopic splinter-like structures called "nanospears" for the targeted delivery of biomolecules such as genes straight to patient cells. These magnetically guided nanostructures could enable gene therapies that are safer, faster and more cost-effective.

15 Mar 2018

Of ‘miracles’ and money: Why hemophilia drugs are so expensive

When Landon Morris was diagnosed with hemophilia shortly after birth, his mother, Jessica Morris, was devastated. "It was like having your dreams — all the dreams you imagined for your child — just kind of disappear," she recalled.

8 Mar 2018

Study unravels novel cellular and molecular pathway that regulates innate, adaptive immunity

Researchers at Brigham and Women's Hospital have discovered a new cellular and molecular pathway that regulates CD4+ T cell response--a finding that may lead to new ways to treat diseases that result from alterations in these cells.

24 Feb 2018