Hemophilia A News and Research

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Gene therapy shows promising effect against blood clots, study states

A new study conducted in the Children's Hospital Of Philadelphia (CHOP) reported positive results in a phase 1/2 clinical trial for the inherited bleeding disorder hemophilia B.

8 Dec 2017

NIH grant supports research on cellular mechanisms governing longevity

The MDI Biological Laboratory has announced that Aric Rogers, Ph.D., has received a grant of $455,000 over two years from the National Institute on Aging, one of the institutes of the National Institutes of Health, for research on the cellular mechanisms governing longevity.

30 Nov 2017

FDA approves new drug to treat bleeding in hemophilia A patients

The U.S. Food and Drug Administration today approved Hemlibra (emicizumab-kxwh) to prevent or reduce the frequency of bleeding episodes in adult and pediatric patients with hemophilia A who have developed antibodies called Factor VIII (FVIII) inhibitors.

17 Nov 2017

FDA permits marketing of first electric stimulation device to reduce opioid withdrawal symptoms

Today, the U.S. Food and Drug Administration granted a new indication to an electric stimulation device for use in helping to reduce the symptoms of opioid withdrawal.

16 Nov 2017

New category of immunotherapy appears safe to use in patients with both cancer and HIV

A new category of immunotherapies called checkpoint inhibitors that has been highly effective against many different cancers appears safe to use in patients with both advanced malignancies and HIV, a population excluded from earlier trials of such therapies, according to an early-phase trial.

7 Nov 2017

FDA advisory committee vote 16-0 for first even gene therapy for rare blindness

A panel of advisors to the United States Food and Drug Administration (FDA) has voted unanimously in favor of an experimental gene therapy to treat patients with a rare kind of hereditary blindness.

16 Oct 2017

New microcapsules represent promising vectors for application of genome-editing tools

Researchers from Tomsk Polytechnic University jointly with their colleagues from St. Petersburg, Hamburg and London have conducted a study in the course of which it was found out that polymer and hybrid silica-coated microcapsules are more efficient in genome-editing when applying CRISPR-Cas9 system.

6 Oct 2017

Pfizer announces launch two new digital innovations to help people living with hemophilia

Pfizer Inc. today announced the launch of Hemocraft, a modification of the popular video game Minecraft, and the HemMobile Striiv Wearable, a custom wristband, to help people living with hemophilia better approach hemophilia education and activity tracking in the US.

26 Aug 2017

Shire partners with MicroHealth to support secure care monitoring tool for hemophilia patients with inhibitors

Shire plc, the biotech leader in rare diseases, today announced a collaboration with MicroHealth to support a free and secure care monitoring tool for hemophilia A and B patients with inhibitors and their care teams.

26 Aug 2017

Researchers investigate genetic mutations in sperm cells to help prevent birth defects

It's about preventing birth defects. Associate pharmaceutical sciences professor Wenfeng An and his team at South Dakota State University are investigating mobile DNA segments, known as L1s, in sperm cells with the goal to treat families at risk for genetic mutations before they conceive.

10 Aug 2017

FDA approves new combo drug Mavyret for adults with Hepatitis C infection

Yesterday the U.S. Food and Drug Administration gave its approval to Mavyret (glecaprevir and pibrentasvir) for the treatment of adults with chronic hepatitis C infection. These patients are infected with the Hepatitis C virus (HCV) of genotypes 1-6 and are those that have not developed liver cirrhosis or those who have developed mild cirrhosis. The approval for Mavyret was given to AbbVie Inc.

3 Aug 2017

Gene-therapy administered through skin transplants could enable treatment of many diseases

A research team based at the University of Chicago has overcome challenges that have limited gene therapy and demonstrated how their novel approach with skin transplantation could enable a wide range of gene-based therapies to treat many human diseases.

3 Aug 2017

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