00:00:00 Introduction by Guy Alba, Chairman of the Board of ELA International

Adrenoleucodystrophy (ALD) – Adrenomyeloneuropathy (AMN)

00:08:44 Florian Eichler – Dynamics of gene therapy and disease biology in ALD

00:40:40 Elise Yazbeck – Care for children with n ALD and the latest updates on NEXUS study

00:55:20 Troy Lund – Hematopoietic stem cell transplant for adult ALD patients

01:18:00 Wolfgang Köhler – Haematopoietic stem cell transplantation in adult patients with ALD

01:42:13 Fanny Mochel – Update on Leriglitazone in adult ALD patients

Pelizaeus-Merzbacher disease (PMD), other hypomyelinating leukodystrophies and Alexander disease

00:00:00 Nicole Wolf – Basis of hypomyelinating leukodystrophies

00:08:40 Paul Tesar – Mode of action of the PMD antisense oligonucleotides (ASOs)

00:26:21 Nicole Wolf – Update on PMD and the ongoing Ionis ORBIT trial

00:40:18 Davide Tonduti – Alexander’s disease: from prediction to treatment – prognostic markers and the Zilganersen trial

00:59:00 Amanda Nagy – ASOs for TUBBA4 : an N=1 experiment

01:22:20 Vivi Heine – Update on a human myelinating spheroid platform for dissecting leukodystrophy pathogenesis

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