FDA approves Waskyra gene therapy for Wiskott-Aldrich Syndrome. Rare disorder affects 1 in 250,000 live male births. Hope for patients.
A rare genetic disorder called WAS primarily impacts males, with approximately 1 in 250,000 live male births being affected. A groundbreaking therapy has brought significant hope to those suffering from this condition. The development was announced in Rome, Italy, on December 10, 2025 by Fondazione Telethon.
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