Hemophilia A News and Research

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Over £800 million to support improved healthcare delivery and breakthroughs in new treatments

Patients up and down the country are set to benefit from innovative new treatments and improved delivery of health and care services following significant funding to support ground-breaking experimental medicine research and advance the UK's response to patient safety challenges.

14 Oct 2022

Researchers receive $9 million grant to study how ‘jumping genes’ influence Alzheimer's disease

Scientists have identified a handful of gene mutations that cause or contribute to the onset of Alzheimer's disease.

11 Oct 2022

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

MOFs could help improve the delivery of genetic cargo into target cells

Hybrid material helps ferry genetic cargo to target cells. An emerging type of material called a metal-organic framework(MOF) could help improve the delivery of genetic material for treating disease.

10 Apr 2022

Immunobridging study shows immunogenicity in adolescents receiving SARS-CoV-2 mRNA and inactivated vaccines

A recent study assessed the reactogenicity and immunogenicity induced by SARS-CoV-2 vaccines in adolescent individuals.

14 Mar 2022

Researchers secure $12 million grant to develop improved therapies for hemophilia

An Indiana University School of Medicine researcher will lead a multi-institute effort toward improved therapies for hemophilia with help from a $12 million grant from the National Heart Lung and Blood Institute.

8 Mar 2022

World's largest genetic study reveals fresh details about severe Covid-19

The world's largest study of the genetics of critical Covid-19, involving more than 57,000 people, has revealed fresh details about some of the biological mechanisms behind the severe form of the disease.

7 Mar 2022

Dexamethasone administration at a late time point could enhance transgene expression

Dexamethasone, a glucocorticoid with anti-inflammatory and immunosuppressive effects, can transiently increase the expression of therapeutic genes delivered using adeno-associated virus (AAV) vectors.

31 Jan 2022

Study determines minimally effective dose of clinical candidate gene therapy vector to treat hemophilia A

Researchers determined the minimally effective dose (MED) of a clinical candidate gene therapy vector for treating hemophilia A.

16 Dec 2021

What does the future look like for bleeding disorders?

In this interview, we speak to Assad Haffar, the Medical and Humanitarian Aid Director at the World Federation of Hemophilia (WFH).

23 Nov 2021

TXA drug has minimal impact in controlling blood loss in severely injured trauma patients

A recent study found that the drug tranexamic acid has minimal impact in controlling blood loss in severely injured trauma patients at the standard doses given, according to researchers at Washington University School of Medicine in St. Louis.

21 Nov 2021

New trial demonstrates stable coagulation factor VIII in hemophilia A patients after gene therapy

A novel gene therapy for hemophilia A led to sustained expression of the clotting factor those patients lack, resulting in a reduction – or in some cases complete elimination – of painful and potentially life-threatening bleeding events, according to a new study led by researchers at Children's Hospital of Philadelphia.

18 Nov 2021

New technology can pave the way for development of long-acting drugs

The technology, consisting of a designed variant of a natural occurring protein called super albumin, can pave the way for the development of long-acting drugs. This can have major implications for individuals suffering from hemophilia.

21 Jul 2021

Why we may never know whether the $56,000-a-year Alzheimer’s drug actually works

The Food and Drug Administration's approval in June of a drug purporting to slow the progression of Alzheimer's disease was widely celebrated, but it also touched off alarms.

12 Jul 2021

New approach to gene therapy uses common pain reliever to fight against genetic diseases

Researchers have developed a new approach to gene therapy that leans on the common pain reliever acetaminophen to force a variety of genetic diseases into remission.

14 Jun 2021

Researchers discover new way to improve the subcutaneous administration of mRNA

If not before, then certainly since the first messenger RNA (mRNA) vaccines to combat the SARS CoV2 virus were approved in Germany, mRNA has become a recognized term even outside scientific circles.

4 May 2021

Immune tolerance in hemophilia can be modulated by B cell activating factor

A group of scientists have just made a key discovery that could prevent and eradicate immune responses that lead to treatment failure in about one-third of people with severe hemophilia A.

20 Apr 2021

CHOP researchers identify key target responsible for treatment failure in patients with hemophilia A

Researchers at Children's Hospital of Philadelphia have identified a key target that may be responsible for treatment failure in about 30% of patients with hemophilia A.

15 Apr 2021

New taxonomic classification of non-skeletal rare congenital disorders with impact on bone physiology

Thanks to major progress in the understanding and management of rare congenital diseases and syndromes, many patients with these rare disorders are now living longer lives.

13 Jan 2021

New clinical practice guidelines on diagnosis, management of von Willebrand Disease

The American Society of Hematology (ASH), the International Society on Thrombosis and Haemostasis (ISTH), National Hemophilia Foundation (NHF), and World Federation of Hemophilia (WFH) have developed joint clinical practice guidelines on the diagnosis and management of von Willebrand Disease (VWD), the world's most common inherited bleeding disorder. The guidelines were published today in Blood Advances.