1. From Barts and the London School of Medicine and Dentistry (K.J.P.), Guy’s and St. Thomas’ NHS Foundation Trust (B.M.), and the Centre for Haematology, Imperial College London (M. Laffan), London, University Hospital Southampton, Southampton (S.R.), University Hospitals Birmingham NHS Foundation Trust, Birmingham (W.L.), and Cambridge University Hospitals NHS Foundation Trust, Cambridge (E.S.) — all in the United Kingdom; and BioMarin Pharmaceutical, Novato (N.M., C.B.R., M. Li, W.Y.W.), and private...