Congenital, Hereditary, and Neonatal Diseases and Abnormalities: News and Research on Congenital, Hereditary, and Neonatal Diseases and Abnormalities

MeSH tree: C16

Medications

FDA approves Waskyra for Wiskott-Aldrich syndrome

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).

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