FDA approves 1st gene therapy for Wiskott-Aldrich syndrome

Ella Jeffries / beckershospitalreview - The FDA has approved Waskyra (etuvetidigene autotemcel), the first gene therapy for Wiskott-Aldrich syndrome, a rare and life-threatening immune disorder. Waskyra is indicated for pediatric patients 6 months and older, as well as adults with Wiskott-Aldri…

AI Summary: In a landmark decision, the FDA approved Waskyra – the first gene therapy for Wiskott‐Aldrich syndrome – offering new hope for patients with this rare and life‐threatening immune disorder. The agency’s flexible approach in handling rare disease treatments marks a turning point in precision medicine.

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