About the Authors

Nicola Specchio

Roles Writing – review & editing

nicola.specchio@gmail.com

Affiliation Department of Neuroscience, Bambino Gesù Children’s Hospital, IRCCS, Rome, Italy

Paul Gissen

Roles Writing – review & editing

Affiliation NIHR Biomedical Research Centre, UCL Great Ormond Street Institute of Child Health, London, United Kingdom

Emily de los Reyes

Roles Writing – review & editing

Affiliation Department of Pediatric Neurology, Nationwide Children’s Hospital, Columbus, Ohio, United States of America

Andrew Olaye

Roles Conceptualization, Writing – review & editing

Current address: UK Orchard Therapeutics Limited, London, United Kingdom

Affiliation BioMarin Europe Ltd, London, United Kingdom

Charlotte Camp

Roles Writing – review & editing

Affiliation BioMarin Europe Ltd, London, United Kingdom

Tristan Curteis

Roles Formal analysis, Writing – original draft

Affiliation Costello Medical, Manchester, United Kingdom

Annabel Griffiths

Roles Conceptualization, Writing – original draft, Writing – review & editing

Affiliation Costello Medical, Cambridge, United Kingdom

Thomas Butt

Roles Conceptualization, Writing – review & editing

Affiliation BioMarin Europe Ltd, London, United Kingdom

Jessica Cohen-Pfeffer

Roles Writing – review & editing

Affiliation BioMarin Pharmaceutical Inc., Novato, California, United States of America

Peter Slasor

Roles Validation, Writing – review & editing

Affiliation BioMarin Pharmaceutical Inc., Novato, California, United States of America

Zlatko Sisic

Roles Writing – review & editing

Current address: Batten Disease Family Association, London, United Kingdom

Affiliation BioMarin Europe Ltd, London, United Kingdom

Mohit Jain

Roles Writing – review & editing

Affiliation BioMarin Europe Ltd, London, United Kingdom

Angela Schulz

Roles Writing – review & editing

Affiliation Department of Pediatrics, Children’s Hospital, University Medical Center Hamburg-Eppendorf, Hamburg, Germany

Competing Interests

PG, NS: No competing interests to declare; AS: Received compensation from BioMarin Europe as per clinical trial agreement; EdlR: Research grants from Amicus Therapeutics, BioMarin Pharmaceutical Inc., Giving Back Fund; Consultancy: BioMarin Pharmaceuticals Inc.; AO, CC, TB, and MJ: Employees and shareholders of BioMarin Europe; ZS: Employee of BioMarin Europe; TC and AG: Employees of Costello Medical; JCP and PS: Employees and shareholders of BioMarin Pharmaceutical Inc. This does not alter our adherence to PLOS ONE policies on sharing data and materials.