A Padua moment for factor VIII gene therapy

considered to be strongly activating lesions, experienced long-term survival.This raises the tantalizing possibility that some patients with PTPN11 mutations and low DNA methylation can safely avoid HSCT.Lastly, the entire study was observational in nature and performed outside of a formal clinical trial.The first riskstratified clinical trial in JMML using DNA methylation and NGS is currently underway in the United States (ClinicalTrials.govidentifier: NCT05849662), but results are not expected until 2028 at the earliest.Until then, the data presented from this well-characterized cohort with long-term follow-up will lead to changes in how pediatric oncologists around the world conceptualize this enigmatic disease.