Hemophilia B News and Research

RSS

In Massachusetts, proposed Medicaid cuts put kids’ health care at risk

The U.S Senate’s plan to replace Obamacare, now in limbo, would cut funding for Medicaid and other health insurance subsidies by more than $1 billion a year within five years.

18 Jul 2017

Novel therapy decreases incidence of bleeding by 87% in hemophilia A patients with inhibitors

An international team of hematologists including Guy Young, MD, of Children's Hospital Los Angeles, has found that in patients with hemophilia A with inhibitors, a novel therapy called emicizumab, decreases incidence of bleeding episodes by 87%.

10 Jul 2017

Abbott introduces Sekisui CP3000 fully automated coagulation system for accurate test results

Today, Abbott announced that the Sekisui CP3000 coagulation system, a fully automated analyzer that tests bleeding and clotting function in blood, is now available in Europe, the Middle East, and Asia Pacific.

23 Jun 2017

International team to set how gene therapies in hemophilia should be measured for effectiveness

An international team involving dozens of stakeholders from patients and policy makers to payers and government agencies are getting together to set how gene therapies in hemophilia should be measured for effectiveness.

20 Jun 2017

Altered virus could help more patients to become eligible for human gene therapy trials

For many patients, participating in gene therapy clinical trials isn't an option because their immune system recognizes and fights the helpful virus used for treatment.

13 Jun 2017

Scripps scientists make important advance in HIV vaccine design

Scientists at The Scripps Research Institute (TSRI) have made another important advance in HIV vaccine design. The development was possible thanks to previous studies at TSRI showing the structures of a protein on HIV’s surface, called the envelope glycoprotein. The scientists used these structures to design a mimic of the viral protein from a different HIV subtype, subtype C, which is responsible for the majority of infections worldwide.

29 May 2017

Sangamo Therapeutics and Pfizer team up for development of Hemophilia A gene therapy

Sangamo Therapeutics, Inc. and Pfizer Inc. today announced an exclusive, global collaboration and license agreement for the development and commercialization of gene therapy programs for Hemophilia A, including SB-525, one of Sangamo's four lead product candidates, which Sangamo expects will enter the clinic this quarter.

11 May 2017

Novo Nordisk introduces new web-based portal that helps patients track their hemophilia treatment

Novo Nordisk today announced the availability of HemaGo™ XChange, a web-based portal that allows people with hemophilia to connect their circle of care to their treatment and bleed information, promoting collaboration and discussion regarding the management of their bleeding disorder.

From Novo Nordisk 13 Apr 2017

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