Fondazione Telethon ETS' Waskyra has become the first gene therapy in the US for patients with Wiskott-Aldrich syndrome (WAS), a rare and life-threatening inherited disease. The Italian non-profit ...
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 ...
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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 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 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 ...
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 ...
The FDA has issued its stamp of approval to a new, cell-based option to treat Wiskott-Aldrich syndrome (WAS), marking the first therapy of its kind for the rare disease and making Italy’s Fondazione ...
The U.S. Food and Drug Administration has approved Waskyra (etuvetidigene autotemcel), the first cell-based gene therapy for the treatment of Wiskott-Aldrich syndrome (WAS). Waskyra is indicated for ...
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 ...
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