1. Great Ormond Street Hospital for Children National Health Service Foundation Trust, London, United Kingdom;
2. School of Pharmacy, and
3. Camden and Islington National Health Service Foundation Trust, London, United Kingdom; and
4. UCL Genetics Institute, University College London, London, United Kingdom;
5. Unité Mixte de Recherche 1137, Institut National de la Santé et de la Recherche Médicale, Université Paris Diderot, Paris, France