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.

Adeno-associated virus: The gene therapy revolution faces manufacturing and safety hurdles

Recombinant adeno-associated virus (rAAV)-based genetic applications to treat human diseases.

5 Apr 2024

Breakthrough gene therapy method utilizes bird retrotransposon for safe gene insertion

The recent approval of a CRISPR-Cas9 therapy for sickle cell disease demonstrates that gene editing tools can do a superb job knocking out genes to cure hereditary disease.

20 Feb 2024

New collaborative proposes strategies to address extreme blood unavailability in rural settings

Billions of people live in parts of the world that are so remote from the nearest hospital facility with a functioning blood bank that they are termed “blood deserts.” Researchers from Brigham and Women’s Hospital, a founding member of the Mass General Brigham healthcare system, and Harvard Medical School have spearheaded a new global collaborative to address the pressing issue of extreme blood unavailability in rural settings and propose short-term strategies to address the urgent need for blood.

13 Feb 2024

Early intervention crucial for faster back pain recovery

Low back pain is a major cause of disability around the globe, with more than 570 million people affected.

22 Jan 2024

Hemoglobin-like protein plays an important role in the development of the heart, study finds

In a landmark study led by the University of Maryland School of Medicine, researchers discovered for the first time that a certain kind of protein similar to hemoglobin, called cytoglobin, plays an important role in the development of the heart.

15 Dec 2023

Liver trigger holds the key to boosting gene therapy success

Indiana University School of Medicine researchers have uncovered vital insights regarding a liver trigger that blocks an undesired immune response from gene therapy, surprisingly resulting in the activation of specific immune cells, despite the liver's typical role in suppressing immune responses.

7 Dec 2023

Silencing galectin 1 protein shrinks liver cancer tumors in mice

Researchers at UC Davis Comprehensive Cancer Center have shown that inhibiting a specific protein using gene therapy can shrink hepatocellular carcinoma (HCC) in mice.

30 Nov 2023

Gene therapy's double-edged sword: Breakthrough treatments face manufacturing and efficacy

Research explores the advancements, challenges, and economic implications of gene therapy for various diseases, including inherited blood disorders, malignancies treated with CAR-T cells, and diseases targeted by in vivo AAV vectors. The review highlights the transformative potential of gene therapies while acknowledging the complexities in manufacturing, safety, efficacy, and long-term durability.

12 Nov 2023

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

Hemophilia News and Research

Further Reading

Adeno-associated virus: The gene therapy revolution faces manufacturing and safety hurdles

Breakthrough gene therapy method utilizes bird retrotransposon for safe gene insertion

New collaborative proposes strategies to address extreme blood unavailability in rural settings

Early intervention crucial for faster back pain recovery

Hemoglobin-like protein plays an important role in the development of the heart, study finds

Liver trigger holds the key to boosting gene therapy success

Silencing galectin 1 protein shrinks liver cancer tumors in mice

Gene therapy's double-edged sword: Breakthrough treatments face manufacturing and efficacy

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

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

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

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

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

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

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

Researchers discover a previously unknown mechanism that drives aging

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

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

First transfusions of lab-grown blood

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

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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