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.

ISTH introduces new global education initiative in gene therapy for hemophilia

The International Society on Thrombosis and Haemostasis is pleased to announce the official launch of Gene Therapy in Hemophilia: An ISTH Education Initiative.

8 Jul 2019

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

New pragmatic study launched to understand the effectiveness of new type 2 diabetes drug

HealthCore, Inc., the outcomes research subsidiary of Anthem, Inc. and Novo Nordisk Inc., which manufacturers the once weekly GLP-1 receptor agonist Ozempic (semaglutide), are working together to launch the first of its kind pragmatic study to understand the benefits of semaglutide as compared with all other available diabetes drugs in a real-world pragmatic clinical trial for Type 2 diabetes - one of the nation's fastest growing chronic diseases.

17 Oct 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

Scientists uncover new strategy to kill tumors without harming healthy cells

Scientists at Scripps Research have uncovered a new strategy to kill tumors, including some triple-negative breast cancers, without harming healthy cells, a discovery that could lead to more ways to treat tumors while reducing side effects.

26 Jul 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

Study finds that several existing anti-cancer drugs may work in part by binding to RNA

Bolstering the notion that RNA should be considered an important drug-discovery target, scientists at Scripps Research have found that several existing, FDA-approved anti-cancer drugs may work, in part, by binding tightly to RNA, the regulators of the basic activities of life within cells.

30 Jun 2018

New study finds way to help treat alcohol addiction and ease withdrawal symptoms

According to a new study by Scripps Research scientists, activating a receptor found in brain cells may help treat alcohol addiction and ease some withdrawal symptoms. The researchers studied the effects of activating the receptor in alcohol-addicted rats and reported the results today in the journal eNeuro.

27 Jun 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