FDA Approves First Gene Therapy for WAS. First Gene Therapy from a Non-Profit
amarcheschi Friday, December 12, 2025
Summary
The FDA has approved the first gene therapy treatment for Wiskott-Aldrich syndrome, a rare and severe genetic disorder that affects the immune system. The treatment involves modifying a patient's own blood stem cells to correct the genetic defect responsible for the condition.
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Summary
fda.gov