Early gene therapy results in Wiskott-Aldrich syndrome promising

Sunday, December 6, 2015 - 22:21 in Health & Medicine

Researchers reported promising preliminary outcomes for the first four children enrolled in a US gene therapy trial for Wiskott-Aldrich syndrome (WAS), a life-threatening genetic blood and immune disorder. All four have improved between nine and 24 months following treatment. Since undergoing treatment, none have experienced bleeding events or severe WAS-related infections. All four have experienced improvements in immunologic symptoms and variable improvements in platelet count.

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