FDA approves Waskyra for Wiskott-Aldrich syndrome
The U.S. Food and Drug Administration has approved Waskyra (etuvetidigene autotemcel) as the first cell-based gene therapy for the treatment of Wiskott-Aldrich syndrome (WAS).
Dec 16, 2025
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Hemic and Lymphatic Diseases: News and Research on Blood Coagulation Disorders, Inherited
MeSH tree: C15.378.100.100
Sustained benefit seen for etranacogene dezaparvovec in hemophilia B
For men with hemophilia B, receipt of gene therapy comprising an infusion of etranacogene dezaparvovec results in sustained endogenous factor IX expression and low annualized bleeding rates over five years, according to a ...
Dec 10, 2025
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Engineered albumin-fused FIX extends hemophilia B treatment half-life beyond standard 3–4 day window
After blood vessel damage, effective blood clotting is essential to halt bleeding. However, this process is inefficient in some individuals due to hereditary factors. Hemophilia B, for example, results from a deficiency in ...
Sep 29, 2025
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Genomics study establishes new rare disease diagnosis framework
A study in China covering 42,703 families affected by rare diseases across 32 provincial regions of China has established a new diagnosis framework for rare diseases. It offers new hope to millions of patients struggling ...
Sep 2, 2025
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Liver organoid generates organ-specific blood vessels for the first time
Scientists from Cincinnati Children's and colleagues based in Japan report achieving a major step forward in organoid technology: producing liver tissue that grows its own internal blood vessels.
Jun 25, 2025
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Hemophilia B gene therapy demonstrates long-term success
A gene transfer approach to treating the bleeding disorder hemophilia B remains safe and effective long-term, as scientists from St. Jude Children's Research Hospital and University College London today report thirteen years ...
Jun 12, 2025
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Discovery could improve treatment for people with hemophilia A
Researchers from the Centenary Institute have uncovered a reason why some people with hemophilia A develop resistance to their critical treatment, paving the way for the development of more effective therapies. The study ...
May 28, 2025
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Efanesoctocog alfa not cost-effective for prophylaxis in hemophilia A
For U.S. patients with severe hemophilia A, prophylaxis with efanesoctocog alfa is neither conventionally nor distributionally cost-effective, according to a study published online April 22 in the Annals of Internal Medicine.
Apr 28, 2025
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FDA approves Qfitlia to cut bleeding episodes in patients with hemophilia
The U.S. Food and Drug Administration has approved Sanofi's Qfitlia (fitusiran) for routine prophylaxis to prevent or reduce the frequency of bleeding episodes in adult and pediatric patients 12 years of age and older with ...
Apr 3, 2025
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Saliva activates coagulation in patients with hemophilia A, study finds
A recent study led by MedUni Vienna provides new insights into the mechanisms of coagulation in people with hemophilia A, the most common form of hemophilia. The research team was able to show that saliva contains special ...
Jan 14, 2025
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