🏅 FDA Orphan Designation
Waskyra
etuvetidigene autotemcel
Manufacturer: Fondazione Telethon ETS
Indicated for:
Wiskott-Aldrich syndromeOrphan
FDA-Approved Indications (1)
Wiskott-Aldrich syndromeOrphan Designation
treatment of pediatric patients aged 6 months and older and adults with Wiskott-Aldrich Syndrome (WAS) who have a mutation in the WAS gene and for whom hematopoietic stem cell transplantation (HSCT) i…
Indications & Usage
treatment of pediatric patients aged 6 months and older and adults with Wiskott-Aldrich Syndrome (WAS) who have a mutation in the WAS gene and for whom hematopoietic stem cell transplantation (HSCT) is appropriate and no suitable human leukocyte antigen (HLA)-matched related stem cell donor is available
💙 Support Programs
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Fondazione Telethon ETS
Where shown, WAC (Wholesale Acquisition Cost) is an estimate only — your actual cost depends on your insurance plan. Always verify pricing with your pharmacy or insurer.
Medical disclaimer: This information is for educational purposes only. Always consult a qualified healthcare provider before making treatment decisions. Data sourced from FDA and current as of our last update.