The rate of severe infections decreased from 2 infections to 0.2 infections per PYO after treatment with etuvetidigene autotemcel. The Food and Drug Administration (FDA) has approved Waskyra ...
The U.S. Food and Drug Administration has approved Waskyra (etuvetidigene autotemcel) as the first cell-based gene therapy for the treatment of Wiskott-Aldrich syndrome (WAS). Waskyra is indicated for ...
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 ...
Waskyra is an ex vivo gene therapy using autologous CD34+ stem cells transduced with a lentiviral vector encoding the WAS gene. The therapy reduces severe bleeding and infections in Wiskott-Aldrich ...
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FDA Approves Waskyra for Wiskott-Aldrich Syndrome
TUESDAY, Dec. 16, 2025 (HealthDay News) -- The U.S. Food and Drug Administration has approved Waskyra (etuvetidigene autotemcel) as the first cell-based gene therapy for the treatment of ...
The US FDA has approved the first ever cell-based gene therapy to treat Wiskott-Aldrich syndrome (WAS). The gene therapy, Waskyra (etuvetidigene), can be used to treat adults with WAS who have a ...
The FDA on Tuesday approved etuvetidigene autotemcel (Waskyra) as the first cell-based gene therapy for patients 6 months and older with Wiskott-Aldrich syndrome, a rare primary immunodeficiency. The ...
WAS affects almost exclusively males, with an estimated incidence of 1 in 250,000 live male births. The therapy represents a major scientific and clinical achievement, offering new hope for patients ...
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