News about "Wiskott-Aldrich Syndrome (WAS)"

EMA Recommends EU Approval for Waskyra to Treat Wiskott-Aldrich Syndrome

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


 

 

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