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

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

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

First transfusions of lab-grown blood

First transfusions of lab-grown blood

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

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

Study elucidates mechanism that is crucial for making hemophilia therapy effective

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

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

MOFs could help improve the delivery of genetic cargo into target cells

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

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

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

Dexamethasone administration at a late time point could enhance transgene expression

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

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

What does the future look like for bleeding disorders?

What does the future look like for bleeding disorders?

TXA drug has minimal impact in controlling blood loss in severely injured trauma patients

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

Why we may never know whether the $56,000-a-year Alzheimer’s drug actually works

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

New approach to gene therapy uses common pain reliever to fight against genetic diseases

Immune tolerance in hemophilia can be modulated by B cell activating factor

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

CHOP researchers identify key target responsible for treatment failure in patients with hemophilia A

Spark Therapeutics launches gene therapy clinical trial for late-onset Pompe disease

Spark Therapeutics launches gene therapy clinical trial for late-onset Pompe disease

New taxonomic classification of non-skeletal rare congenital disorders with impact on bone physiology

New taxonomic classification of non-skeletal rare congenital disorders with impact on bone physiology