Haemophilia B News and Research

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Monthly prophylactic fitusiran injections effective in reducing bleeds in patients with hemophilia A or B

Monthly prophylactic injections of fitusiran are effective in reducing bleeds in patients with hemophilia A or B, according to randomized controlled trials publishing simultaneously in The Lancet and The Lancet Haematology journals.

30 Mar 2023

New project develops more efficient treatment approaches for cardiovascular diseases

Within the framework of a European collaborative research project, Oliver Müller, professor at the Faculty of Medicine at Kiel University and Consultant at the Department of Internal Medicine III, University Medical Center Schleswig-Holstein, Campus Kiel, will receive 1.4 million Euros for the project "CardioReGenix: Development of Next-Generation Gene Therapies for Cardiovascular Disease".

21 Dec 2018

Researchers uncover genes that play a key role in the inactivation of X chromosomes

In cell biology, men and women are unequal: men have an X chromosome, while women have two. How can we get around this difference?

19 Dec 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

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 editing within a living person for the first time

Genetic editing has been performed in the laboratory until now. Living cells within Petri-dishes have been put under the microscope while gene editing tools such as CRISPR were used to cut, nick and replace parts of the DNA or genome that is defective with bits of DNA that is correct in its sequence. Now doctors in California have attempted to edit cells within a living person.

19 Nov 2017

Contaminated blood scandal that infected thousands in 70’s and 80’s being investigated

In the 1970’s and 80’s thousands of people were infected with hepatitis C and HIV due to transfusion of contaminated blood that contained these viruses. A spokesperson for Theresa May, James Slack, today said that she, her health secretary Jeremy Hunt and the MPs sat together to announce a full review and inquiry into this disaster.

12 Jul 2017

Octapharma uses G:BOX Chemi XX6 imager to accurately characterise large therapeutic proteins

Syngene, a world-leading manufacturer of image analysis solutions, is pleased to announce its G:BOX Chemi XX6 multi-application-functional imager is being utilised by scientists at major human protein R&D company, Octapharma Biopharmaceuticals GmbH in Germany for analysing a range of large proteins.

From Syngene 17 Mar 2017

Non-adherence to treatment increases risk of major bleeds in young people with haemophilia, study finds

Almost one in five young people with haemophilia in England and Wales are increasing the risk of major bleeds by not taking adequate treatment.

25 Jan 2017

Elite Team GB cyclist launches 'Little Bleeders' to tackle inactivity in young haemophilia patients

Young Ambassadors, healthcare professionals and some of the UK’s elite athletes have today joined together to launch Little Bleeders – a foundation tackling inactivity in boys and young men living with haemophilia, head-on across the UK.

21 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

An Introduction to Specialty Pharmaceuticals

In this interview, News Medical talks to David Moran, Managing Director of Clinigen SP about specialty pharmaceuticals.

From Clinigen Group 29 Aug 2016

Coagadex now available for rare bleeding disorder patients in the UK

Bio Products Laboratory, Limited (BPL) today announced that Coagadex is now available for patients in the UK.

7 Jun 2016

European Medicines Agency approves first-ever treatment for hereditary factor X deficiency

Bio Products Laboratory, Limited (BPL) today announced that the European Medicines Agency has granted marketing authorisation for Coagadex.

31 Mar 2016

New €5.6 million project aims to develop cell-based haemophilia A therapy

Loughborough University is working with a team of international partners on a new €5.6 million project to develop a cell based haemophilia A therapy.

25 Jan 2016

Improved gene therapy treatment shows promise in mice with cystic fibrosis

An improved gene therapy treatment can cure mice with cystic fibrosis (CF). Cell cultures from CF patients, too, respond well to the treatment. Those are the encouraging results of a study presented by the Laboratory for Molecular Virology and Gene Therapy at KU Leuven, Belgium.

17 Nov 2015

Simple, low risk blood test can detect foetal blood group and genetic conditions in unborn babies

Research into a simple, accurate and low risk blood test that can detect foetal blood group, sex, and genetic conditions in unborn babies has been published in the international scientific journal, Clinical Chemistry.

9 Nov 2015

Approval to start trial of Videregen's replacement trachea technology

The INSPIRE consortium, led by Videregen Ltd, has received approval from the MHRA1 to start a UK clinical trial with its tissue engineered replacement trachea.

3 Nov 2015

New report on different treatment strategies for haemophilia published

Numerous long-term randomized controlled trials (RCTs) have been conducted to investigate the long-term, factor concentrate-based treatment of patients with severe haemophilia A or B, despite the rareness of the diseases and the lack of incentives for pharmaceutical companies.

14 Jul 2015

Apitope's ATX-F8-117 granted FDA Orphan Drug Designation for treatment of haemophilia A patients

Apitope, the drug discovery and development company focused on disease-modifying treatments that reinstate immune tolerance, announced today that pre-clinical product candidate ATX-F8-117 has been granted Orphan Drug Status by the US Food and Drug Administration for the prevention or treatment of inhibitors in haemophilia A patients with inhibitors or at the risk of producing inhibitors.

9 Jun 2015