Hemophilia A News and Research

RSS

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

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

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 '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

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

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 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

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

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

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

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

Shielding stem cells with novel biomaterial can reduce heart attack damage

Bioengineers and surgeons from Rice University and Baylor College of Medicine (BCM) have shown that shielding stem cells with a novel biomaterial improves the cells' ability to heal heart injuries caused by heart attacks.

18 Aug 2020

New articles focus on recent developments in the field of health preference research

Value in Health, the official journal of ISPOR-;the professional society for health economics and outcomes research, announced today the publication of a series of articles focused on recent developments in the field of health preference research.

10 Aug 2020

A new cell-based gene therapy approach to better treat patients with hemophilia A

In a new study from the Wake Forest Institute for Regenerative Medicine researchers have developed an optimized cellular platform for delivering Factor 8 to better treat patients with hemophilia A.

28 Jul 2020

GlobalData: Hematology tests can be a prognostic marker to predict severity of COVID-19 patients

With the rapid increase in the number of COVID-19 cases across India, identifying the critically ill COVID-19 patients in the early stage of infection can help in assessing the risk stratification and clinical management.

21 Jul 2020

2021 health plans granted leeway to limit consumers’ benefit from drug coupons

Without medication to manage her plaque psoriasis, Jennifer Brown's face, scalp, trunk and neck periodically become covered in painful red, flaky patches so dry they crack and bleed.

6 Jul 2020

Hemophilia A News and Research

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

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

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

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

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

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

Study elucidates mechanism that is crucial for making hemophilia therapy effective

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

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

What does the future look like for bleeding disorders?

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

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

Providing personalized care for bleeding disorders

Providing personalized care for bleeding disorders

Shielding stem cells with novel biomaterial can reduce heart attack damage

New articles focus on recent developments in the field of health preference research

A new cell-based gene therapy approach to better treat patients with hemophilia A

GlobalData: Hematology tests can be a prognostic marker to predict severity of COVID-19 patients

2021 health plans granted leeway to limit consumers’ benefit from drug coupons

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

Latest News