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.

New CRISPR-based gene-editing tool could lead to better treatments for genetic disorders

A new CRISPR-based gene-editing tool has been developed which could lead to better treatments for patients with genetic disorders.

29 Sep 2023

Once-weekly ALTUVIIIO® approved in Japan as a new class of factor VIII therapy for hemophilia A

The Japanese Ministry of Health, Labor, and Welfare (MHLW) has granted marketing authorization for ALTUVIIIO.

From Sanofi 25 Sep 2023

New method of insulin delivery lowers the risk of hypoglycemia compared to current diabetes treatments

Insulin production has, for the last 50 or so years, come with some risks to the patient. Even so, the medication is lifesaving for the estimated 537 million adults living with diabetes worldwide, with that number expected to grow.

15 Jun 2023

This panel will decide whose medicine to make affordable. Its choice will be tricky.

Catherine Reitzel's multiple sclerosis medication costs nearly $100,000 a year. Kris Garcia relies on a drug for a blood-clotting disorder that runs $10,000 for a three-day supply. And Mariana Marquez-Farmer would likely die within days without her monthly $300 vial of insulin.

25 May 2023

UWaterloo expert helps develop database repository to manage treatment for hemophilia patients

Today is World Hemophilia Day under the theme ‘Access for all: prevention of bleeds as the global standard of care’. Dr. Andrea Edginton, Hallman Director at the University of Waterloo School of Pharmacy, uses her research to help pharmacists, doctors and nurses manage treatment for hemophilia patients that has, in turn, helped these patients lead a better quality of life.

18 Apr 2023

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 'cutting edge' hemophilia treatment is a victory for patients, Tulane doctor says

A new treatment that helps people with hemophilia A maintain higher levels of a crucial blood clotting factor with fewer treatments is a victory for patients, according to a new editorial in The New England Journal of Medicine this week by Dr. Cindy Leissinger, director of the Louisiana Center for Bleeding and Clotting Disorders at Tulane University School of Medicine.

27 Jan 2023

Researchers discover a previously unknown mechanism that drives aging

Northwestern University researchers have discovered a previously unknown mechanism that drives aging.

9 Dec 2022

Liver cancer study could help improve the safety of certain gene therapies for hemophilia

Research led by Randal J. Kaufman, Ph.D., has found that misfolded proteins in liver cells contribute to the development of liver cancer, shedding new light on the mysterious origins of one of the world's deadliest diseases.

7 Dec 2022

New CRISPR-based tool can snip out faulty genes and replace them with new ones

Building on the CRISPR gene-editing system, MIT researchers have designed a new tool that can snip out faulty genes and replace them with new ones, in a safer and more efficient way.

24 Nov 2022

First transfusions of lab-grown blood

Two patients in the United Kingdom received two small doses of lab-grown blood samples as part of the RESTORE trial.

9 Nov 2022

Beyond vaccines: emerging technologies capable of unlocking the promise of biologically targeted mRNA therapeutics

Researchers reviewed technology advancements that will unlock the promise of biologically targeted messenger ribonucleic acid (mRNA) therapeutics beyond vaccines.

7 Nov 2022

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