Hemophilia A News and Research

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With federal nod, consumers could lose the boost they get from drug ‘coupons’

Patients who get financial help from drug companies to cover their copayments for prescription drugs could owe a bigger chunk of their costs under a proposed federal rule.

16 Apr 2020

'Living drug factories' could be used to achieve long-term treatment of chronic diseases

One promising way to treat diabetes is with transplanted islet cells that produce insulin when blood sugar levels get too low.

30 Mar 2020

FDA approves immune-modulating therapy for treatment of multiple sclerosis

The U.S. Food and Drug Administration has approved ozanimod, an immune-modulating therapy invented at Scripps Research, for the treatment of adults with relapsing forms of multiple sclerosis.

26 Mar 2020

Dog study suggests that AAV in gene therapy could induce cancer

A gene therapy study in dogs has stirred up old fears that by using AAV in gene therapy, it may be increasing cancer chances.

8 Jan 2020

Study shows safety, long-term beneficial impact of gene therapy for patients with Hemophilia A

A breakthrough gene therapy treatment for Haemophilia A has been shown to offer long-term benefits that have already transformed the lives of 13 men in the UK.

6 Jan 2020

Researchers discover new way to supercharge protein production

Medicines such as insulin for diabetes and clotting factors for hemophilia are hard to synthesize in the lab. Such drugs are based on therapeutic proteins, so scientists have engineered bacteria into tiny protein-making factories.

18 Dec 2019

Inbreeding between royalty led to facial defects

The royal dynasty that ruled huge Spanish and Austrian territories over the last century was called the Habsburgs and were famous for more than their imperial majesty. The “Habsburg jaw”, a facial condition that afflicted these kings and queens, was well known. Now a new study says that this facial dysmorphism was due to inbreeding. The study was published in the Annals of Human Biology.

2 Dec 2019

Mentorship in medical school linked to increased likelihood of fellows specializing in hematology

A survey of U.S. hematology-oncology fellows suggests medical school plays an important role in shaping their interest in pursuing careers in hematology, particularly when students are exposed to hematology and oncology as part of core clerkships in internal medicine and pediatrics.

31 Oct 2019

CDC to fund sickle cell disease surveillance

The Centers for Disease Control and Prevention (CDC) has announced a funding of $1.2 million to different states in order to collect data and information from people suffering from sickle cell disease. The program called the Sickle Cell Data Collection program or SCDC is at present functional in two states - California and Georgia. With the new funds coming in, nine states would be able to collect and send population based information on the health of persons living with sickle cell disease.

27 Sep 2019

Hemophilia much more prevalent than previously thought

More than 1,125,000 men around the world have the inherited bleeding disorder of hemophilia, and 418,000 of those have a severe version of the mostly undiagnosed disease, says a new study led by McMaster University researchers.

9 Sep 2019

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

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