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News about "Wiskott-Aldrich Syndrome (WAS)"
FDA Approves Waskyra Gene Therapy for Wiskott-Aldrich Syndrome
Fondazione Telethon announced that the US FDA has approved Waskyra (etuvetidigene autotemcel), a gene therapy for Wiskott-Aldrich Syndrome (WAS), marking a milestone in both science and clinical care. The approval offers new hope for patients living with this rare immunodeficiency disorder.
Wiskott-Aldrich Syndrome (WAS) | 11/12/2025 | By Akanki | 162
EMA Recommends EU Approval for Waskyra to Treat Wiskott-Aldrich Syndrome
The EMA has recommended EU marketing authorisation for Waskyra (etuvetidigene autotemcel), a gene therapy for patients aged six months and older with Wiskott-Aldrich Syndrome (WAS). The treatment uses a patient’s own modified stem cells to restore functional WAS protein.
Wiskott-Aldrich Syndrome (WAS) | 17/11/2025 | By Dineshwori | 157
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