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WASKYRA (etuvetidigene autotemcel) is a gene therapy for pediatric patients aged 6 months and older and adults with Wiskott-Aldrich Syndrome who have a mutation in the WAS gene. It is indicated for patients for whom hematopoietic stem cell transplantation is appropriate but no suitable HLA-matched related stem cell donor is available.
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en.wikipedia.org shapes more of what AI says about Waskyra than any other source, at 100% of its citations.