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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Hemic and Lymphatic Diseases: News and Research on Wiskott-Aldrich Syndrome
MeSH tree: C15.378.463.960
Stem cells can tailor their role in gene therapy based on the underlying disease, study suggests
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.
Gene therapy may cure rare diseases. But drugmakers have few incentives, leaving families desperate
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.
Jun 21, 2024
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