FDA Approves Waskyra (etuvetidigene autotemcel) Gene Therapy for the Treatment of Wiskott-Aldrich Syndrome

Dec 11, 2025 - 01:00
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December 10, 2025 -- Fondazione Telethon announced today that the U.S. Food and Drug Administration (FDA) has approved the Biologics License Application (BLA) for Waskyra, an ex vivo gene therapy for patients with Wiskott-Aldrich syndrome (WAS), a...

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