First FDA-Approved Gene Therapy for Wiskott-Aldrich Syndrome

This is for families of children (and adults) with genetically confirmed Wiskott-Aldrich syndrome who need a transplant-style path and do not have a suitable HLA-matched related donor — and who may be candidates for labelled US Waskyra one-time autologous gene-modified stem-cell therapy at a qualified treatment centre after mobilisation, apheresis, conditioning, and Telethon / Orphan Therapies logistics. It explains the first FDA-approved gene therapy for WAS, the infection and bleeding rate changes, safety monitoring, how to start a centre referral conversation, and why authorised is not funded — without treating unnamed centres as confirmed, treating this as a walk-in infusion, or importing a US bag. Authorized is not funded.