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large vessel vasculopathy an underrecognized complication in wiskott aldrich syndrome
Original title: “Large vessel vasculopathy: An underrecognized complication in Wiskott-Aldrich syndrome.”
The U.S. Food and Drug Administration today approved Waskyra (etuvetidigene autotemcel), the first cell-based gene therapy for the treatment of Wiskott-Aldrich syndrome (WAS).
Original title: “FDA Approves First Gene Therapy Treatment for Wiskott-Aldrich Syndrome”