Females with Fabry disease often experience diagnostic delays and underappreciated disease burden due to variable disease presentation and progression. The spectrum of disease severity and long-term efficacy of migalastat were assessed in females with Fabry disease and amenable GLA variants. Most had multiorgan involvement at baseline, suggesting significant disease burden. Migalastat treatment preserved renal function, stabilized left ventricular mass, and was associated with a low incidence of Fabry-associated clinical events. These data offer unique insights into treatment for females, historically underrepresented in Fabry disease clinical trials, and support long-term efficacy of migalastat, even in cases of considerable disease severity. (By Dr. Staci Kallish, https://jmg.bmj.com/content/early/2025/09/01/jmg-2024-110596 )