ELA Conference families-scientists 2026 • March 29 • Day 2

00:00 Introduction by Guy Alba, Chairman of the Board of ELA International Basis of genetic 00:08:48 Françoise Piguet – Basis of genetic to understand genetic diseases Aicardi-Goutières Syndrome (AGS), metachromatic leukodystrophy (MLD), Krabbe disease and Zellweger spectrum disorders 00:34:00 Marie‑Louise Frémond – Aicardi-Goutières Syndrome: 20 years after the First Gene Discovery 00:54:54 Caroline Sevin – Care and treatments currently under development for MLD 02:00:16 Carole Linster – Modelling a PEX1 deficiency, from cells to zebrafish, to identify potential therapeutic leads