[{"data":1,"prerenderedAt":-1},["ShallowReactive",2],{"health-studies-list:{\"conditionNormalized\":\"multiple-sulfatase-deficiency-disease\",\"overallStatus\":[\"RECRUITING\",\"AVAILABLE\",\"NOT_YET_RECRUITING\"],\"orderBy\":\"LastUpdateSubmitDate:desc\",\"size\":25,\"offset\":0}":3,"health-study-condition:multiple-sulfatase-deficiency-disease":30},{"pageToken":4,"total":5,"offset":6,"count":5,"results":7},null,2,0,[8,44],{"id":9,"slug":10,"hasResults":11,"nctId":12,"briefTitle":13,"officialTitle":14,"acronym":4,"eligibilityCriteria":15,"healthyVolunteers":11,"sex":16,"minAge":4,"maxAge":4,"enrollmentInfo":17,"targetDuration":4,"studyType":20,"phases":4,"briefSummary":21,"conditions":22,"keywords":4,"overallStatus":31,"whyStopped":4,"lastUpdateSubmitDate":32,"lastUpdatePostDateStruct":33,"startDateStruct":36,"completionDateStruct":38,"leadSponsor":40,"locationsCount":43},"100518911","mps-radico-cohort-radico-mps-100518911",false,"NCT06036693","MPS (RaDiCo Cohort) (RaDiCo-MPS)","Mucopolysaccharidosis Patients in France in the Era of Specific Therapeutics","Inclusion Criteria:\n\n* Confirmed diagnosis of MPS based on clinically relevant enzyme deficiency, with abnormally elevated GAG urinary excretion and\u002For identification of pathogenic mutations.\n* Signed informed consent or parents\u002Fguardian non-opposition for deceased patients (minor or protected major)\n\nThere are no non-inclusion criteria.","ALL",{"count":18,"type":19},1000,"ESTIMATED","OBSERVATIONAL","The goal of this observational study is to characterize the epidemiology and natural history of MPS diseases by building a retrospective and prospective collection of extensive phenotypic data from French MPS patients.",[23,24,25,26,27,28,29,30],"Mucopolysaccharidosis I","Mucopolysaccharidosis II","Mucopolysaccharidosis III","Mucopolysaccharidosis IV","Mucopolysaccharidosis VI","Mucopolysaccharidosis VII","Mucopolysaccharidosis IX","Multiple Sulfatase Deficiency Disease","RECRUITING","2026-02-10",{"date":34,"type":35},"2026-02-11","ACTUAL",{"date":37,"type":35},"2017-12-20",{"date":39,"type":19},"2026-12",{"name":41,"class":42},"Institut National de la Santé Et de la Recherche Médicale, France","OTHER_GOV",23,{"id":45,"slug":46,"hasResults":11,"nctId":47,"briefTitle":48,"officialTitle":48,"acronym":4,"eligibilityCriteria":49,"healthyVolunteers":11,"sex":16,"minAge":4,"maxAge":4,"enrollmentInfo":50,"targetDuration":4,"studyType":20,"phases":4,"briefSummary":52,"conditions":53,"keywords":80,"overallStatus":31,"whyStopped":4,"lastUpdateSubmitDate":95,"lastUpdatePostDateStruct":96,"startDateStruct":98,"completionDateStruct":100,"leadSponsor":102,"locationsCount":105},"100311346","longitudinal-study-of-neurodegenerative-disorders-100311346","NCT03333200","Longitudinal Study of Neurodegenerative Disorders","Inclusion Criteria:\n\n* Any patient with a genetic neurodegenerative disorder\n\nExclusion Criteria:\n\n* none",{"count":51,"type":19},1500,"The purpose of this study is to understand the course of rare genetic disorders that affect the brain. This data is being analyzed to gain a better understanding of the progression of the rare neurodegenerative disorders and the effects of interventions.",[54,55,56,57,58,59,60,61,62,63,64,65,66,67,68,69,70,71,72,73,74,75,76,77,78,79,30],"MLD","Krabbe Disease","ALD","MPS I","MPS II","MPS III","Vanishing White Matter Disease","GM3 Gangliosidosis","PKAN","Tay-Sachs Disease","NP Deficiency","Osteopetrosis","Alpha-Mannosidosis","Sandhoff Disease","Niemann-Pick Diseases","MPS IV","Gaucher Disease","GAN","GM1 Gangliosidoses","Morquio Disease","S-Adenosylhomocysteine Hydrolase Deficiency","Batten Disease","Pelizaeus-Merzbacher Disease","Leukodystrophy","Lysosomal Storage Diseases","Purine Nucleoside Phosphorylase Deficiency",[81,82,83,84,85,86,87,88,89,90,91,92,93,94],"Pediatric","Rare","Neurodegenerative","Genetic","Neurodevelopment","Brain","MRI","Biorepository","NDRD","Longitudinal","Cognitive","Motor","Language","Adaptive behavior","2026-02-04",{"date":97,"type":35},"2026-02-09",{"date":99,"type":35},"2012-01-11",{"date":101,"type":19},"2035-01",{"name":103,"class":104},"University of Pittsburgh","OTHER",1]