FDA Flexibility Leads to First Approved Gene Therapy for Rare Blood & Immune System Disorder
Waskyra is the first FDA-approved gene therapy for the rare disease Wiskott-Aldrich syndrome and the first such approved product from a non-profit applicant, Fondazione Telethon. Data from Waskyra’s pivotal studies were presented this week during the annual meeting of the American Society of Hematology.
