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TrialConditionsStatusPhaseUpdatedMore infoNCT ID
A Global Prospective Observational Registry of Patients With Pompe DiseaseBrain Diseases, Metabolic, Brain Diseases, Metabolic, Inborn, Congenital, Hereditary, and Neonatal Diseases and Abnormalities, Lysosomal Storage Diseases, Nervous System, Nutritional and Metabolic Diseases, brain disorder, central nervous system disorder, disorder of glycogen metabolism, glycogen storage disease II, hereditary disease, inborn carbohydrate metabolic disorder, inborn errors of metabolism, lysosomal storage disease, metabolic disease, nervous system disorderRecruitingNot stated2026-03-10NCT06121011

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OrganizationsLinked trials
Amicus Therapeutics7

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Research sitesCityCountryLinked trials
Aarhus Universitets hospitalAarhus CDenmark1
Azienda Ospedaliero Universitaria Consorziale Policlinico di BariBariItaly5
Cambridge University - Addenbrooke's HospitalCambridgeUnited Kingdom1
Centre of Expertise for muscular diseases and peripheral neuropathies European Reference Network for Rare Neuromuscular DiseasesNaplesItaly1
Cincinnati Children's HospitalCincinnatiUnited States66
Department of Neurosciences Rita Levi Montalcini, University of TorinoTorinoItaly1
Dept of Pediatrics Erasmus MC - Sophia Children's HospitalRotterdamNetherlands1
Duke University Medical CenterDurhamUnited States505
Eginition HospitalAthensGreece1
Emory UniversityAtlantaUnited States262
entrum Medyczne MedykRzeszówPoland1
Great Ormond Street Hospital NHS Foundation TrustLondonUnited Kingdom3
Indiana University, IU Health Physicians NeurologyIndianapolisUnited States1
Laboratory for Muscle Diseases and NeuropathiesLeuvenBelgium1
Lysosomal and Rare Disorders Research and Treatment Center, Inc.FairfaxUnited States6
Medizinische Universitaet WienViennaAustria11
National Hospital For Neurology and NeurosurgeryLondonUnited Kingdom4
NYU Neurogenetics, NYU Langone Medical CenterNew YorkUnited States1
Queen Elizabeth Hospital BirminghamBirminghamUnited Kingdom13
Regional Coordinating Centre for Rare Diseases, university Hospital of Udine, Udine, ItalyUdineItaly1
Royal Free Hospital NHS Foundation TrustManchesterUnited Kingdom1
Ruhr-Universität Bochum im St. Josef-HospitalBochumGermany1
Salford Royal NHS Foundation TrustSalfordUnited Kingdom4
SphinCS, Institute of Clinical Science in LSDHöchheimGermany1
Universitaetsklinikum UlmUlmGermany34