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 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

Over £800 million to support improved healthcare delivery and breakthroughs in new treatments

Patients up and down the country are set to benefit from innovative new treatments and improved delivery of health and care services following significant funding to support ground-breaking experimental medicine research and advance the UK's response to patient safety challenges.

14 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

Innovative Augmented Reality Joint Scanner unveiled at Westmead Children's Hospital to support young people with hemophilia

An Australian-designed, first-of-its-kind Augmented Reality technology has been unveiled today at The Children’s Hospital at Westmead (CHW). The technology will enable young people living with Hemophilia to view the potentially irreversible impact of disabling joint disease.

31 May 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

World's largest genetic study reveals fresh details about severe Covid-19

The world's largest study of the genetics of critical Covid-19, involving more than 57,000 people, has revealed fresh details about some of the biological mechanisms behind the severe form of the disease.

7 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

Hemophilia News and Research

Further Reading

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

Study elucidates mechanism that is crucial for making hemophilia therapy effective

Over £800 million to support improved healthcare delivery and breakthroughs in new treatments

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

Innovative Augmented Reality Joint Scanner unveiled at Westmead Children's Hospital to support young people with hemophilia

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

World's largest genetic study reveals fresh details about severe Covid-19

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

New technology can pave the way for development of long-acting drugs

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

Researchers discover new way to improve the subcutaneous administration of mRNA

Industry Focus eBook - Forensics & toxicology (2nd edition) eBook

Designing fluidic architecture for scalable diagnostic platforms

Evaluating new approaches for rare disease genomics

Expanding access to NMR with the Fourier 80 Duo

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