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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.
Expanded Access Programme | 17/11/2025 | By Dineshwori
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