Hemophilia A News and Research

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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

Novel method using big data tools shows promising routes for research

A group of researchers affiliated to the University of São Paulo - campus Ribeirão Preto, Brazil, used big data tools such as data mining and network analysis to develop a method to help scientists and companies identify technological routes, trends and partnerships in any knowledge area. For this purpose, it collected information from patent databases around the world.

3 Sep 2018

Subcutaneous injection of new protein helps treat hemophilia more effectively

Patients with hemophilia bleed spontaneously or following trauma. The most common sites of bleeding are into joints, but bleeds can also happen into any tissue in the body including the muscles, brain, or eyes.

31 Aug 2018

Promising data supports development of AAVhu37-based gene therapy for hemophilia A

Gene therapy using an optimized adeno-associated virus to deliver the human factor VIII gene to cynomolgus macaques showed a substantial increase in hFVIII expression and no detectable antibody response for 30 weeks in some animals.

21 Aug 2018

RCSI and Bayer collaborate to improve treatments for people with hemophilia

RCSI and Bayer have today announced a research collaboration that aims to improve treatments for people with severe haemophilia.

9 Aug 2018

Researchers use genome editing to reduce cholesterol levels in animal model

Using genome editing to inactivate a protein called PCSK9 effectively reduces cholesterol levels in rhesus macaques, a species of monkey, according to researchers from the Perelman School of Medicine at the University of Pennsylvania.

10 Jul 2018

ABPI associated with NHS for 70 years in pharmaceutical research

In this special year, both the NHS and the Association of the British Pharmaceutical Industry are celebrating our respective 70th anniversaries. The histories of these two organizations have been inextricably linked since 1948 and our relationship with the NHS continues to go from strength to strength.

25 Jun 2018

LogicBio team to present data on preclinical effectiveness of GeneRide platform

LogicBio Therapeutics, Inc., a genetic medicine company founded to develop safe medicines with lasting therapeutic benefit for children with life-threatening diseases, today announced it will be presenting data on the company's technology and robust preclinical pipeline at the annual meeting of the American Society of Gene & Cell Therapy at the Hilton Chicago, from May 16-19, 2018.

14 May 2018

Breakthrough study promises to bring gene-editing technology much closer to therapeutic reality

Imagine a future where a guided biomachine put into your body seeks out defective gene sequences in each cell and edits in the correct information with precision accuracy.

13 Apr 2018

UCLA-developed nanospears could make gene therapies safer, faster and more cost-effective

UCLA scientists have developed a new method that utilizes microscopic splinter-like structures called "nanospears" for the targeted delivery of biomolecules such as genes straight to patient cells. These magnetically guided nanostructures could enable gene therapies that are safer, faster and more cost-effective.

15 Mar 2018

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