Hemophilia News and Research

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Hemophilia is a rare, inherited bleeding disorder in which your blood doesn’t clot normally. If you have hemophilia, you may bleed for a longer time than others after an injury. You also may bleed internally, especially in your knees, ankles, and elbows. This bleeding can damage your organs or tissues and, sometimes, be fatal.

Study elucidates mechanism that is crucial for making hemophilia therapy effective

Hemophilia A is the most common severe form of hemophilia. It affects almost exclusively males. The disease can usually be treated well, but not for all sufferers.

27 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

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

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

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

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

Spark Therapeutics launches gene therapy clinical trial for late-onset Pompe disease

Pompe disease is a hereditary genetic disorder caused by a deficiency of acid alpha-glucosidase leading to build-up of glycogen in the lyosomes, which then causes cell damage in various tissues, in particular the heart, the muscles, the liver and the nervous system.

3 Feb 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.

13 Jan 2021

NIH-funded study to test emergency treatment for devastating stroke in Memphis

The University of Tennessee Health Science Center's Stroke Team is joining researchers from more than 100 hospitals worldwide to conduct a National Institutes of Health (NIH)-funded research study called FASTEST that will test the safety and efficacy of a medication called recombinant factor Vlla to prevent hemorrhage expansion in patients with strokes caused by bleeding in the brain - intracerebral hemorrhage strokes.

19 Nov 2020

Providing personalized care for bleeding disorders

An interview with Dr. Carmen Escuriola-Ettingshausen MD, Mörfelden-Walldorf, Germany on providing personalized care for bleeding disorders.

From Takeda 21 Sep 2020

Providing personalized care for bleeding disorders

An interview with Dr. Carmen Escuriola-Ettingshausen MD, Mörfelden-Walldorf, Germany on providing personalized care for bleeding disorders.

From Takeda 21 Sep 2020

Hemophilia News and Research

Further Reading

Study elucidates mechanism that is crucial for making hemophilia therapy effective

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

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

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

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

Dexamethasone administration at a late time point could enhance transgene expression

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

What does the future look like for bleeding disorders?

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

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

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

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

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

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

Spark Therapeutics launches gene therapy clinical trial for late-onset Pompe disease

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

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

NIH-funded study to test emergency treatment for devastating stroke in Memphis

Providing personalized care for bleeding disorders

Providing personalized care for bleeding disorders

Achieving and maintaining ISO 27001 and BS 10012 certifications

Expanding research and clinical options for children with cancer

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

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