The strong safety record of SFX-01 in adult mice, including NS models, together with solid evidence of its tolerability in humans (Clack et al, 2025; Howell et al, 2019; Long et al, 2024; Zolnourian et al, 2024; Zolnourian et al, 2020), and its ability to reverse Shp2-driven myeloproliferative disease, make it a promising candidate for clinical trials aimed at treating disorders caused by mutations that activate this phosphatase. Here, PTPN11 is linked to myeloproliferative disorder.