Recent Advances in Extended Half-Life Products, Nonfactor Replacement Therapies, and Gene Therpy for the Treatment of Hemophilia.

Authors

Prakash Vishnu, David M Aboulafia

Published in

Clinics in laboratory medicine. Volume 45. Issue 4. Pages 657-673. Epub Aug 13, 2025.

Abstract

Hemophilia, a rare X-linked hereditary bleeding disorder, is characterized by deficiency of coagulation factors. Symptoms range from spontaneous joint and muscle bleeds to life-threatening hemorrhage, for which patients require frequent infusion of coagulation factors, profoundly impacting their quality of life. Novel therapeutics and advances in gene therapy now offer patients long-term disease control and improved quality of life. Here, we focus on important developments in the use of extended half-life factor products, non-factor replacement therapies, and gene therapy. We also highlight the mechanism of action, clinical efficacy, and safety of these newer approaches.

PMID:
41161982
Bibliographic data and abstract were imported from PubMed on 30 Oct 2025.

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