Hemophilia B News and Research

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Of ‘miracles’ and money: Why hemophilia drugs are so expensive

When Landon Morris was diagnosed with hemophilia shortly after birth, his mother, Jessica Morris, was devastated. "It was like having your dreams — all the dreams you imagined for your child — just kind of disappear," she recalled.

8 Mar 2018

Study unravels novel cellular and molecular pathway that regulates innate, adaptive immunity

Researchers at Brigham and Women's Hospital have discovered a new cellular and molecular pathway that regulates CD4+ T cell response--a finding that may lead to new ways to treat diseases that result from alterations in these cells.

24 Feb 2018

Scientists discover adult endothelial stem cells capable of generating fully functional blood vessels

The proper function of blood vessels is essential to life: blood vessels are responsible for transporting oxygen-rich red blood cells, nutrients, and immune cells throughout the body, to name just a few functions. Defects in blood vessels can correspondingly lead to a variety of life-threatening diseases.

15 Feb 2018

Biomedical engineers develop miniature self-sealing wound model

Biomedical engineers have developed a miniature self-sealing model system for studying bleeding and the clotting of wounds. The researchers envision the device as a drug discovery platform and potential diagnostic tool.

8 Feb 2018

Sangamo and Pfizer partner for development of new zinc finger protein gene therapy to treat ALS

Sangamo Therapeutics, Inc. and Pfizer Inc. today announced a collaboration for the development of a potential gene therapy using zinc finger protein transcription factors (ZFP-TFs) to treat amyotrophic lateral sclerosis (ALS) and frontotemporal lobar degeneration (FTLD) linked to mutations of the C9ORF72 gene.

8 Jan 2018

Strategies to prevent and treat acute chest syndrome in children with sickle cell disease

Acute chest syndrome (ACS), a potentially severe lung complication of sickle cell disease, increases a child's risk of respiratory failure, chronic lung disease, and prolonged hospitalization if not recognized early and treated effectively.

5 Jan 2018

Groundbreaking gene therapy trial brings cure for hemophilia closer

A 'cure' for hemophilia is one step closer, following results of a groundbreaking gene therapy trial led by the NHS in London.

14 Dec 2017

New, more easily administered therapies offer benefits for bleeding and clotting disorders

In three studies being presented today during the 59th American Society of Hematology (ASH) Annual Meeting and Exposition in Atlanta, researchers report remarkable benefits from new, more easily administered therapies for bleeding and clotting disorders.

11 Dec 2017

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.