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Reflecting on the trial Mojsov remembers the therapeutic effect was immediately evident

Gollob MH, Seger JJ, Gollob TN, Tapscott T, Gonzales O, Bachinski L, Roberts R (2001) Novel PRKAG2 mutation responsible for the genetic syndrome of ventricular preexcitation and conduction system disease with childhood onset and absence of cardiac hypertrophy

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Future studies expanding patient-derived brain organoids and PBMC analyses to capture genetic, immunological, and disease-stage heterogeneity, particularly during acute disease, would further improve the translational relevance and guide precision clinical trials