1. Department of Gastroenterology, UCSF Benioff Children's Hospital Oakland Oakland California USA
2. Department of Child NeurologyJustus‐Liebig University Gieβen Germany
3. Department of Paediatrics, Fondazione MBBM San Gerardo Hospital Monza Italy
4. San Raffaele Telethon Institute for Gene Therapy (SR‐TIGET)IRCCS San Raffaele Scientific Institute Milan Italy
5. Department of Endocrinology and Metabolic Medicine, Salford Royal Hospital NHS Foundation Trust Salford UK
6. Department of Metabolic Diseases, São João Hospital Porto Portugal
7. BioMarin Pharmaceutical Inc. Novato California USA