FDA approves first gene therapy for rare blood 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.

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Top Story 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. Sponsored Moving Beyond an Insurance-Based Framework for Healthcare Presented by UberDoc CEO Sean Kearney breaks down how his company wants to simplify booking appointments. Provide more control for physicians and empower patients to understand the cost for care they receive. Read more to learn how UberDoc is changing the game.

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