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Wiskott–Aldrich syndrome
Trends
- 1Gene therapy shows lasting benefit in Wiskott–Aldrich syndrome▼Gene therapy shows sustained benefit in Wiskott–Aldrich syndrome
New findings indicate gene therapy delivers sustained benefits for patients with Wiskott–Aldrich syndrome, a rare inherited immune disorder. The treatment, which replaces a faulty gene, reportedly continues working over extended follow-up, offering an alternative to bone marrow transplant. The development is drawing attention from clinicians and families affected by the condition.