Tag: Wiskott-Aldrich syndrome
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Wiskott-Aldrich Gene Therapy: EU EMA Recommends Waskyra for WAS
What is Wiskott-Aldrich syndrome and the promise of gene therapy Wiskott-Aldrich syndrome (WAS) is a rare, inherited immune disorder caused by mutations in the WAS gene. The condition typically presents in infancy or early childhood with a mix of immune dysfunction, small blood cells (thrombocytopenia), eczema, and an increased risk of infections and autoimmune problems.…
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EU Considers First Gene Therapy for Wiskott-Aldrich Syndrome: Waskyra
EU Moves Toward First Gene Therapy for Wiskott-Aldrich Syndrome The European Medicines Agency (EMA) has recommended granting marketing authorization in the European Union for Waskyra (etuvetidigene autotemcel), a gene therapy designed to treat people aged 6 months and older with Wiskott-Aldrich syndrome (WAS) who carry mutations in the WAS gene. If approved, Waskyra would become…
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First EU Approval Consideration for WAS Gene Therapy: Waskyra (etuvetidigene autotemcel)
What is Waskyra and Who Could Benefit? The European Medicines Agency (EMA) has recommended granting marketing authorisation in the European Union for Waskyra (etuvetidigene autotemcel) to treat people aged 6 months and older with Wiskott-Aldrich syndrome (WAS) who carry a mutation in the WAS gene. Waskyra is a one-time, autologous gene therapy that aims to…