Disease

WAS associated disorder1, 2

Gene

WAS

Inheritance XL
Current Treatments Waskyra (etuvetidigene autotemcel (OTL‑103) - autologous CD34+ HSPC transduced with a lentiviral vector encoding WAS, granulocyte colony-stimulating factor (G-CSF), Bone marrow transplant (hematopoietic stem cell transplantation (HSCT)), Antibiotic prophylaxis, immunoglobulin replacement
Evidence for Treatment expert opinion
Non-Molecular Testing Availability yes
Non-Molecular Test complete blood count1
ClinGen https://actionability.clinicalgenome.org/ac/Pediatric/ui/stg2SummaryRpt?doc=AC011
Disease Frequency (per 100,000) 0.1751, 3
Genes Included in Disease Frequency WAS
Experimental / Investigational Therapies https://clinicaltrials.gov/ct2/results?cond=Wiskott-Aldrich+Syndrome&term=&cntry=&state=&city=&dist=
Last Updated: March 5, 2026
RX Genes
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