✉news HealthMedicine first seen 22 h ago, last 10 h ago, peak #19
Gene therapy shows sustained benefit in Wiskott–Aldrich syndrome
New findings indicate that gene therapy continues to provide sustained benefit for patients with Wiskott–Aldrich syndrome, a rare inherited immune disorder. The results suggest the one-time treatment can offer long-lasting improvement in immune function, offering hope to families affected by the condition, which historically has been managed with transplants or supportive care.
Why now: The publication of new clinical evidence showing long-term effectiveness of a therapy for a rare disease is drawing attention from the medical community and affected families.
Wiskott–Aldrich syndromegene therapy
Evidence
- Gene therapy shows sustained benefit in Wiskott–Aldrich syndrome · 2 Minute Medicine
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