FDA approves Waskyra for Wiskott-Aldrich syndrome
The U.S. Food and Drug Administration has approved Waskyra (etuvetidigene autotemcel) as the first cell-based gene therapy for the treatment of Wiskott-Aldrich syndrome (WAS).
Dec 16, 2025
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MeSH tree: C20.673.627
The U.S. Food and Drug Administration has approved Waskyra (etuvetidigene autotemcel) as the first cell-based gene therapy for the treatment of Wiskott-Aldrich syndrome (WAS).
Dec 16, 2025
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RCCS San Raffaele Scientific Institute in Milan researchers have discovered that hematopoietic stem cells (HSCs) adapt their lineage commitment during gene therapy based on the underlying genetic disease.
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Robin Alderman faces an agonizing reality: Gene therapy might cure her son Camden's rare, inherited immune deficiency. But it's not available to him.
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A medication used to treat multiple sclerosis (MS) also has a beneficial effect on the composition of the intestinal flora, according to researchers from the University of Basel and the University Hospital Basel. Conversely, ...
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