August 19, 2025BiomedicinesOpen Access

Curative Therapies for Hemophilias and Hemoglobinopathies in Adults: Immune, Gene, and Stem Cell Approaches in a Global Context

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Discussion

Review outlines immune-based therapies and gene modification for hemophilias and hemoglobinopathies, indicating global implementation challenges.

Recent advances in therapies for hemophilias and hemoglobinopathies demonstrate durable efficacy, enhancing quality of life for adults.
Immune-based therapeutics like emicizumab and viral vector-mediated gene therapies show potential for curative intervention.
Barriers to implementation include high costs and regulatory differences across regions, affecting access to therapies.
Ethical concerns persist, highlighting the need for coordinated action to ensure equitable access to these innovative treatments.

Bangolo et al. (2025) studied this question.

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Review outlines immune-based therapies and gene modification for hemophilias and hemoglobinopathies, indicating global implementation challenges.