Hemophilia News and Research

RSS

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.

Advances in chemistry and delivery are taking oligonucleotide medicines beyond rare diseases

A review in Cell Reports Medicine examines how advances in oligonucleotide chemistry and delivery have established ASOs and RNA interference therapies as a class of genetic medicines, with 21 oligonucleotides approved at the time of writing. It also identifies extrahepatic delivery, safety, target validation, individualized treatment, and data quality as priorities as the field expands into common diseases and explores AI-assisted development.

24 Sep 2026

Advances in X chromosome inactivation open therapeutic opportunities for X-linked genetic disorders

A new review explores how advances in understanding X chromosome inactivation (XCI) are creating potential therapeutic opportunities for a range of X-linked genetic disorders, including Rett syndrome, Fabry disease, Duchenne muscular dystrophy, hemophilia, and others.

3 Aug 2026

Engineered proteins track gene expression in living primate brains

Gene therapy has been successfully used to treat a number of diseases, including immune deficiencies, hereditary blindness, hemophilia and, recently, Huntington's disease, a fatal neurological disorder.

27 Feb 2026

Advances in hemophilia gene therapy bring hope for pediatric patients

In the past three years, gene therapy has reshaped what's possible in hemophilia treatment for patients 18 and older. But a key question remains: How soon will these advances reach children?

18 Dec 2025

Why soybean oil may fuel weight gain

Researchers investigate how soybean oil may disrupt fat metabolism to potentially increase the risk of obesity.

4 Dec 2025

New sequencing method reveals how transposons move and shape the genome

Cornell researchers have found that a new DNA sequencing technology can be used to study how transposons move within and bind to the genome.

22 Nov 2025

International experts publish guidelines on the administration of gene therapy for hereditary hearing loss

Up to 60% of congenital and early-onset hearing loss is caused by genetic mutations in an inherited gene, and gene therapy has recently emerged as a potential treatment option.

23 Oct 2025

New gene-editing method can correct many disease-causing mutations in mammalian cells

Some genetic disorders-such as cystic fibrosis, hemophilia and Tay Sachs disease-involve many mutations in a person's genome, often with enough variation that even two individuals who share the same disorder might have a different combination of mutations.

23 Oct 2025

Researchers design factor IX variants for short- and long-term Hemophilia B management

After blood vessel damage, effective blood clotting is essential to halt bleeding. However, this process is inefficient in some individuals due to hereditary factors. Hemophilia B, for example, results from a deficiency in coagulation factor IX (FIX), which can lead to prolonged bleeding after injuries or surgery.

30 Sep 2025

Scientists create liver organoids with functional blood vessels

Liver organoids with proper blood vessel networks have been successfully produced, as reported by researchers from Institute of Science Tokyo and Cincinnati Children's Hospital Medical Center.

17 Jul 2025

New cellular entry pathway improves gene therapy outcomes

Scientists from the Centenary Institute and the University of Sydney have made a landmark discovery that could lead to safer and more effective gene therapies for a range of serious genetic disorders including Duchenne muscular dystrophy, Pompe disease and hemophilia.

15 Jul 2025

New study supports non-surgical approach for select cases of acute subdural hematoma

Acute subdural hematoma (ASDH) is a common brain lesion resulting from traumatic brain injuries and is associated with high mortality and morbidity.

27 Jun 2025

Gene therapy shows long-term success in hemophilia B patients

A gene transfer approach to treating the bleeding disorder hemophilia B remains safe and effective long-term, as scientists from St. Jude Children's Research Hospital and University College London today report thirteen years of follow-up data.

12 Jun 2025

New gene editor evoCAST enables precise insertion of complete genes

Ask scientists what gene editing tool is most needed to advance gene therapy, and they'd probably describe a system that's now close to realization in the labs of Samuel Sternberg at Columbia University Vagelos College of Physicians and Surgeons and David Liu at the Broad Institute of MIT and Harvard.

15 May 2025

Marstacimab approved to treat patients aged 12 years and above (weighing at least 35 kg) with hemophilia A or B

The Medicines and Healthcare products Regulatory Agency (MHRA) has approved the medicine marstacimab (Hympavzi) to prevent or reduce bleeding in patients 12 years of age and older weighing at least 35 kg with hemophilia A and B.

23 Apr 2025

The role of saliva in Hemophilia A coagulation

A recent study led by MedUni Vienna provides new insights into the mechanisms of coagulation in persons with haemophilia A, the most common form of haemophilia. The research team was able to show that saliva contains special vesicles that trigger rapid coagulation of the blood of haemophilic patients. The results, which were recently published in the renowned scientific journal Blood, contribute significantly to a better understanding of the disease.

15 Jan 2025

Concizumab shows promising results for hemophilia patients

For people with the bleeding disorder hemophilia, life can seem like one continuous state of alertness. If they accidentally cut their finger while chopping vegetables, they have to immediately administer the clotting factor protein that their body either lacks or does not produce enough of.

2 Dec 2024

Transposons play essential role in early human development

A critical transition in early human development is regulated not by our own genes, but by DNA elements called transposons that can move around the genome, Sinai Health researchers have found.

16 Oct 2024

Study reveals life-changing impact of gene therapy for hemophilia B patients

Adults with hemophilia B saw their number of bleeding episodes drop by an average of 71 percent after a single infusion of gene therapy, according to the results of an international Phase III clinical trial published today in the New England Journal of Medicine by researchers from the University of Pennsylvania Perelman School of Medicine and a multicenter group of investigators.

25 Sep 2024

Breakthrough in hemophilia A treatment: Sobi's ALTUVOCT receives EU marketing authorization

Sobi® today announced the European Commission has granted Marketing Authorization for ALTUVOCT™ (efanesoctocog alfa), for the treatment and prevention of bleeds and perioperative prophylaxis in hemophilia A. ALTUVOCT is a high-sustained factor VIII replacement therapy for all ages and any disease severity.

22 Jun 2024