Exploring concurrent validity of the CLN2 Clinical Rating Scale: Comparison to PedsQL using cerliponase alfa clinical trial data

• Nicola Specchio ,

  Roles Writing – review & editing

  * E-mail: nicola.specchio@gmail.com

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

  https://orcid.org/0000-0002-8120-0287

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• Paul Gissen,

  Roles Writing – review & editing

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

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• Emily de los Reyes,

  Roles Writing – review & editing

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

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• Andrew Olaye,

  Roles Conceptualization, Writing – review & editing

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

  Affiliation BioMarin Europe Ltd, London, United Kingdom

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• Charlotte Camp,

  Roles Writing – review & editing

  Affiliation BioMarin Europe Ltd, London, United Kingdom

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• Tristan Curteis,

  Roles Formal analysis, Writing – original draft

  Affiliation Costello Medical, Manchester, United Kingdom

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• Annabel Griffiths,

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

  Affiliation Costello Medical, Cambridge, United Kingdom

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• Thomas Butt,

  Roles Conceptualization, Writing – review & editing

  Affiliation BioMarin Europe Ltd, London, United Kingdom

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• Jessica Cohen-Pfeffer,

  Roles Writing – review & editing

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

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• Peter Slasor,

  Roles Validation, Writing – review & editing

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

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• Zlatko Sisic,

  Roles Writing – review & editing

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

  Affiliation BioMarin Europe Ltd, London, United Kingdom

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• Mohit Jain,

  Roles Writing – review & editing

  Affiliation BioMarin Europe Ltd, London, United Kingdom

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• Angela Schulz

  Roles Writing – review & editing

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

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