Wiskott-Aldrich Syndrome
Dec 19, 2025
WASKYRA Wins FDA Approval, Marking a Historic Leap for Rare Disease Gene Therapies
In a landmark decision on December 10, 2025, the FDA approved WASKYRA (etuvetidigene autotemcel), marking a transformative milestone in the treatment of Wiskott-Aldrich syndrome—a rare, life-threatening genetic blood disorder. This approval represents not only a significant advancement in gene therapy but also a hi...
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