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TrialConditionsStatusPhaseUpdatedMore infoNCT ID
A Long-Term Observational Study of Patients With FucosidosisBrain 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, fucosidosis, hereditary disease, inborn carbohydrate metabolic disorder, inborn errors of metabolism, lysosomal storage disease, metabolic disease, nervous system disorderRecruitingNot stated2026-09-17NCT07615400
MT2013-31: Allo HCT for Metabolic Disorders and Severe OsteopetrosisAbnormalities, Multiple, Adrenoleukodystrophy, Neonatal, Bone Diseases, Developmental, Brain Diseases, Metabolic, Brain Diseases, Metabolic, Inborn, Congenital Abnormalities, Congenital, Hereditary, and Neonatal Diseases and Abnormalities, Digestive System Diseases, Female Urogenital Diseases, Female Urogenital Diseases and Pregnancy Complications, Hereditary Central Nervous System Demyelinating Diseases, Hurler syndrome, Krabbe disease, Leukoencephalopathies, Lipid Metabolism Disorders, Lipid Metabolism, Inborn Errors, Lysosomal Storage Diseases, Nervous System, Male Urogenital Diseases, Mannosidase Deficiency Diseases, Mucopolysaccharidosis I, Neurobehavioral Manifestations, Neurologic Manifestations, Nutritional and Metabolic Diseases, Peroxisomal Disorders, Refsum Disease, Infantile, Skin and Connective Tissue Diseases, Sulfatidosis, Urogenital Diseases, X-linked disease, Zellweger spectrum disorders, alpha-mannosidosis, alpha-methylacyl-CoA racemase deficiency, aspartylglucosaminuria, bone disorder, brain disorder, central nervous system disorder, connective tissue disorder, cutaneous mucinosis, d-bifunctional protein deficiency, demyelinating disease, fucosidosis, hereditary disease, inborn carbohydrate metabolic disorder, inborn errors of metabolism, inherited neurodegenerative disorder, intellectual disability, kidney disorder, leukoencephalopathy, diffuse hereditary, with spheroids 1, liver disorder, lysosomal lipid storage disorder, lysosomal storage disease, metabolic disease, metachromatic leukodystrophy, mitochondrial neurogastrointestinal encephalomyopathy, mucopolysaccharidosis, mucopolysaccharidosis type 2, mucopolysaccharidosis type 6, mucopolysaccharidosis type 7, musculoskeletal system disorder, nervous system disorder, osteochondrodysplasia, osteopetrosis, osteosclerosis, peroxisomal acyl-CoA oxidase deficiency, sphingolipidosis, urinary system disorderActive, not recruitingPhase 22026-01-07NCT02171104