Hemophilia A News and Research

RSS

UMass Amherst study highlights unmet drug needs of patients with rare diseases

A new analysis by health policy researchers at the University of Massachusetts Amherst who studied the effectiveness of the 1983 Orphan Drug Act finds that current incentives "are not sufficiently stimulating orphan drug development" by pharmaceutical companies, and patients with rare diseases and conditions still have unmet needs. Further, barriers to ethical and timely access remain.

16 Feb 2017

Salk scientists use mRNA to successfully treat hemophilia B in mice

FedEx, UPS, DHL--when it comes to sending packages, choices abound. But the most important delivery service you may not have heard of mRNA.

16 Feb 2017

Plant-based drug for hemophilia shows promise in animal models

People with hemophilia require regular infusions of clotting factor to prevent them from experiencing uncontrolled bleeding. But a significant fraction develop antibodies against the clotting factor, essentially experiencing an allergic reaction to the very treatment that can prolong their lives.

13 Feb 2017

New gene therapy for Pompe disease may replace currently available treatments

After decades investigating a rare, life-threatening condition that cripples the muscles, Duke Health researchers have developed a gene therapy they hope could enhance or even replace the only FDA-approved treatment currently available to patients.

27 Jan 2017

AAV-based gene therapy to treat liver disorders advances into human testing

Liver-directed gene therapy delivered using adeno-associated viral (AAV) vectors to treat diseases such as hemophilia have advanced into human testing.

30 Dec 2016

Researchers working to develop pill for inherited bleeding disorders

Motivated by the tribulations of hemophilia patients and their families, researchers funded by the National Institute of Biomedical Imaging and Bioengineering are working to develop a pill to treat this serious inherited bleeding disorder.

23 Dec 2016

Hemophilia B patients produce near-normal levels of clotting factor IX after gene therapy, study shows

Researchers are reporting the highest and most sustained levels to date of the essential blood-clotting factor IX in patients with the inherited bleeding disorder hemophilia B.

4 Dec 2016

Penn researchers use CRISPR/Cas9 gene targeting approach to treat hemophilia B in mice

CRISPR/Cas9, a powerful genome editing tool, is showing promise for efficient correction of disease-causing mutations.

1 Dec 2016

UT Austin researchers develop new oral capsule for treating hemophilia patients

In the near future, hemophiliacs could be able to treat their disease by simply swallowing a capsule.

29 Nov 2016

ASGCT seeks to educate public and policy-makers on fundamentals of gene editing

On Nov. 21, 2016, the American Society of Gene & Cell Therapy released Therapeutic Gene Editing: an ASGCT White Paper, intended to provide policy-makers, patient advocates, and the interested public with the necessary background information in anticipation of an upcoming consensus report on human gene editing from the National Academy of Sciences and National Academy of Medicine (NAS), expected to be released in early 2017.

24 Nov 2016

New European study emphasizes need to enhance standard of haemophilia care in real life

Swedish Orphan Biovitrum AB (publ) announces the results from a new European study that assessed the efficacy of haemophilia care in real life.

14 Nov 2016

Two leading non-profit organizations sign strategic affiliation to hasten drug development

The Scripps Research Institute and the California Institute for Biomedical Research - two leading non-profit research organizations - today announced the signing of a strategic affiliation that combines the two organizations into a new biomedical research entity with the tools and know-how to rapidly translate its scientific discoveries into life-saving medicines for the public benefit.

20 Oct 2016

New virtual reality games create better patient-experience for kids during infusions

As a nurse clinician in the comprehensive hemophilia treatment center at Nationwide Children's Hospital for nearly 30 years, Charmaine Biega, RN, has watched her patients endure hundreds of needle sticks for infusions and other procedures which can mean tears, frustration, wiggling and - in some cases - lifelong anxiety about the medical system and treatments that patients with hemophilia need to survive.

5 Oct 2016

Scientists link malfunctioning molecular pathways to specific heart abnormalities in SCA

Patients with sickle cell anemia (SCA) develop heart complications and nearly a quarter die a sudden death. Now, researchers have linked malfunctioning molecular pathways to specific heart anomalies in SCA that result from progressive fibrosis and result in sudden death.

9 Aug 2016

UNC professor identifies research priorities to address VTE in cancer patients

More than 20 percent of all blood clots in veins occur in cancer patients. These clots, also known as venous thromboembolism (VTE), pose serious threats for cancer patients.

14 Jul 2016

TSRI awarded $20 million for first year of precision medicine initiative cohort program

As part of the most ambitious medical research program in the history of American medicine, The Scripps Research Institute (TSRI) has received an initial award of $20 million for its role in a national precision medicine initiative, the National Institutes of Health (NIH) announced today.

8 Jul 2016

Study provides more insight into treatment options for severe hemophilia A

Families of children with severe hemophilia A may want to take a fresh look at treatment options from human plasma. A study published in the New England Journal of Medicine on May 26 showed that participants who received a recombinant therapy— the present standard in the United States — developed antibodies or "inhibitors" to the treatments at almost twice the rate as those whose treatments were made from human plasma.

14 Jun 2016

Scientists use new technique to repair fibrotic liver cells within the organ

Advances in stem cell research have made it possible to convert patients' skin cells into heart cells, kidney cells, liver cells and more in the lab dish, giving researchers hope that one day such cells could replace organ transplantation for patients with organ failure.

3 Jun 2016

SIPPET study may have implications for treatment of patients with severe hemophilia A

SIPPET, a study which involved 42 centers in 14 countries in Europe, North and South America, Africa and Asia was designed to definitively settle the long-debated question whether factor VIII concentrates from different sources (plasma-derived containing VWF or recombinant technology) differ in risk of inhibitor development in previously untreated children (PUPs) with severe hemophilia A.

28 May 2016

Jumping genes may play crucial role in generation of cancer

For more than 50 years, scientists have known of the existence of "jumping genes," strands of DNA material that can move from one location in the genome to another.

11 May 2016