Haemophilia A News and Research

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Hemophilia A is an X-linked, recessive, bleeding disorder caused by a deficiency in the activity of coagulation factor VIII. Affected individuals develop a variable phenotype of hemorrhage into joints and muscles, easy bruising, and prolonged bleeding from wounds. The disorder is caused by heterogeneous mutations in the factor VIII gene which maps to Xq28.

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

Rare Diseases Action Plan for England focuses on providing better care and reducing health inequalities

A new Rare Diseases Action Plan for England will be published today (Tuesday 28 February) that will ensure those living with these conditions receive better care and treatment, fairer access to testing and have continued support.

28 Feb 2023

New milestones for the treatment of hemophilia

Hemophilia A is the most common severe form of hemophilia. It affects almost exclusively the male sex. The disease can usually be treated well, but the medicines known so far do not help all of those affected.

9 Feb 2023

Innovative Augmented Reality Joint Scanner unveiled at Westmead Children's Hospital to support young people with hemophilia

An Australian-designed, first-of-its-kind Augmented Reality technology has been unveiled today at The Children’s Hospital at Westmead (CHW). The technology will enable young people living with Hemophilia to view the potentially irreversible impact of disabling joint disease.

31 May 2022

Study shows safety, long-term beneficial impact of gene therapy for patients with Hemophilia A

A breakthrough gene therapy treatment for Haemophilia A has been shown to offer long-term benefits that have already transformed the lives of 13 men in the UK.

6 Jan 2020

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

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

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

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 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

Haemophilia A News and Research

Further Reading

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

Rare Diseases Action Plan for England focuses on providing better care and reducing health inequalities

New milestones for the treatment of hemophilia

Innovative Augmented Reality Joint Scanner unveiled at Westmead Children's Hospital to support young people with hemophilia

Study shows safety, long-term beneficial impact of gene therapy for patients with Hemophilia A

New project develops more efficient treatment approaches for cardiovascular diseases

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

Subcutaneous injection of new protein helps treat hemophilia more effectively

RCSI and Bayer collaborate to improve treatments for people with hemophilia

Groundbreaking gene therapy trial brings cure for hemophilia closer

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

Gene editing within a living person for the first time

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

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

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

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

New European study emphasizes need to enhance standard of haemophilia care in real life

An Introduction to Specialty Pharmaceuticals

Coagadex now available for rare bleeding disorder patients in the UK

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

Expanding research and clinical options for children with cancer

Examining how pain could play a direct protective role in the gut

From Puberty to Menopause: Clue’s CEO, Audrey Tsang on the Power of Femtech

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