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Italian non-profit set for European gene therapy first in Wiskott-Aldrich syndrome
Fondazione Telethon’s Waskyra (etuvetidigene autotemcel) has been recommended for approval by the CHMP as a treatment for the rare disease.
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Wiskott–Aldrich syndrome (WAS)
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Gene therapy shows promise in Wiskott–Aldrich syndrome
Lentiviral gene therapy reduces infections and bleeding episodes in children with a severe immune disorder Wiskott–Aldrich syndrome (WAS), finds new study.
