Post by Leen Hertens

I'm pleased to announce my very first publication on short-term outcomes in pediatric patients with mild RPE65-related IRD, who were treated with voretigene neparvovec. According to current knowledge, early treatment benefits efficacy. However, consensus on treating mild cases is lacking due to ambiguity in balancing clinical benefits with potential side effects. I would like to sincerely thank dr. David Merle and prof. Katarina Stingl for this collaboration and for including two Belgian patients in this article , as well as prof. dr. Bart Peter LEROY for providing helpful feedback during the writing of this paper and the opportunity for me to be the shared-first author. https://lnkd.in/eRVQ9F68