Condition

Wiskott-Aldrich Syndrome

Treatments and trials tagged Wiskott-Aldrich Syndrome, with the same country and access zoom as New Developments.

All New Developments

Wiskott-Aldrich Syndrome breakthroughs

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FDA ApprovedUnited States

First FDA-Approved Gene Therapy for Wiskott-Aldrich Syndrome

Waskyra (etuvetidigene autotemcel) — one-time gene-modified stem-cell therapy for Wiskott-Aldrich syndrome when no matched related donor is available

FDA approval on 9 December 2025 for Waskyra (etuvetidigene autotemcel) suspension — an autologous hematopoietic stem-cell gene therapy given as a…

This is for families of children (and adults) with genetically confirmed Wiskott-Aldrich syndrome who need a…