Phase 3 — Large-scale testing across thousands of people, comparing the new treatment against current standard treatments.
(37 sites) United States
UCSF Children's Hospital and Research Center at Oakland, Oakland, California University of Minnesota Medical School, Minneapolis, Minnesota Mayo Clinic Childrens Center - PIN, Rochester, Minnesota Children's Medical Center Dallas, Dallas, Texas Lysosomal Rare Disorders Research and Treatment Center, Fairfax, Virginia Argentina
Hospital Universitario Austral, Ciudad Autónoma Buenos Aires, Buenos Aires Hospital de Niños de La Santisima Trinidad, Córdoba, Córdoba Province Australia
Women's and Children's Hospital, North Adelaide, South Australia Royal Melbourne Hospital, Parkville, Victoria Royal Children's Hospital Melbourne - PIN, Parkville, Victoria Brazil
Instituto Fernandes Figueira, Rio de Janeiro, Rio de Janeiro Hospital de Clinicas de Porto Alegre (HCPA) - PPDS, Porto Alegre, Rio Grande do Sul Hospital Pequeno Príncipe, Curitiba Canada
M.A.G.I.C. Clinic Ltd. Metabolics and Genetics in Calgary, Calgary, Alberta University of Alberta Medical Genetics Clinic, Edmonton, Alberta Centre Hospitalier de l'Universite de Montreal-1000 rue Saint-Denis, Montreal, Quebec France
AP-HP - Hôpital Armand Trousseau, Paris India
Amrita Institute of Medical Sciences and Research Centre, Ernākulam, Kerala All India Institute of Medical Sciences (AIIMS) - New Delhi, New Delhi, National Capital Territory of Delhi JK Lone Hospital, Jaipur, Rajasthan Christian Medical College and Hospital, Vellore, Tamil Nadu Italy
Fondazione IRCCS Istituto Neurologico Carlo Besta, Milan Mexico
Hospital Universitario Dr. Jose Eleuterio González, Monterrey, Nuevo León Centenario Hospital Miguel Hidalgo, Aguascalientes Portugal
ULS de Santo António, EPE - Centro Materno Infantil Norte, Porto, Porto District ULS de Santa Maria,EPE - Hospital de Santa Maria - PPDS, Lisbon Spain
Hospital Universitario Vall d'Hebron - PPDS, Barcelona, Barcelona Hospital Infantil Universitario Niño Jesus - PIN, Madrid, Madrid Sweden
Sahlgrenska universitetssjukhuset Östra, Gothenburg, Västra Götaland County Switzerland
Inselspital - Universitätsspital Bern, Bern, Canton of Bern Turkey (Türkiye)
Balcali Hastanesi Saglik Uygulama ve Arastirma Merkezi, Adana, Adana Gazi Universitesi Saglik Arastirma ve Uygulama Merkezi, Çankaya, Ankara Ege Universitesi Tip Fakultesi, Bornova, İzmir United Kingdom
Great Ormond Street Hospital, London, London University College London Hospitals (UCLH), London, Middlesex Royal Manchester Children's Hospital, Manchester