[{"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":104},{"pageToken":4,"total":5,"offset":6,"count":5,"results":7},null,2,0,[8,43],{"id":9,"slug":4,"hasResults":10,"nctId":11,"briefTitle":12,"officialTitle":13,"acronym":4,"eligibilityCriteria":14,"healthyVolunteers":10,"sex":15,"minAge":4,"maxAge":4,"enrollmentInfo":16,"targetDuration":4,"studyType":19,"phases":4,"briefSummary":20,"conditions":21,"keywords":4,"overallStatus":30,"whyStopped":4,"lastUpdateSubmitDate":31,"lastUpdatePostDateStruct":32,"startDateStruct":35,"completionDateStruct":37,"leadSponsor":39,"locationsCount":42},"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":17,"type":18},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.",[22,23,24,25,26,27,28,29],"Mucopolysaccharidosis I","Mucopolysaccharidosis II","Mucopolysaccharidosis III","Mucopolysaccharidosis IV","Mucopolysaccharidosis VI","Mucopolysaccharidosis VII","Mucopolysaccharidosis IX","Multiple Sulfatase Deficiency Disease","RECRUITING","2026-02-10",{"date":33,"type":34},"2026-02-11","ACTUAL",{"date":36,"type":34},"2017-12-20",{"date":38,"type":18},"2026-12",{"name":40,"class":41},"Institut National de la Santé Et de la Recherche Médicale, France","OTHER_GOV",23,{"id":44,"slug":4,"hasResults":10,"nctId":45,"briefTitle":46,"officialTitle":46,"acronym":4,"eligibilityCriteria":47,"healthyVolunteers":10,"sex":15,"minAge":4,"maxAge":4,"enrollmentInfo":48,"targetDuration":4,"studyType":19,"phases":4,"briefSummary":50,"conditions":51,"keywords":78,"overallStatus":30,"whyStopped":4,"lastUpdateSubmitDate":93,"lastUpdatePostDateStruct":94,"startDateStruct":96,"completionDateStruct":98,"leadSponsor":100,"locationsCount":103},"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":49,"type":18},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.",[52,53,54,55,56,57,58,59,60,61,62,63,64,65,66,67,68,69,70,71,72,73,74,75,76,77,29],"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",[79,80,81,82,83,84,85,86,87,88,89,90,91,92],"Pediatric","Rare","Neurodegenerative","Genetic","Neurodevelopment","Brain","MRI","Biorepository","NDRD","Longitudinal","Cognitive","Motor","Language","Adaptive behavior","2026-02-04",{"date":95,"type":34},"2026-02-09",{"date":97,"type":34},"2012-01-11",{"date":99,"type":18},"2035-01",{"name":101,"class":102},"University of Pittsburgh","OTHER",1,""]