[{"data":1,"prerenderedAt":-1},["ShallowReactive",2],{"health-studies-list:{\"conditionNormalized\":\"mucopolysaccharidosis-vii\",\"overallStatus\":[\"RECRUITING\",\"AVAILABLE\",\"NOT_YET_RECRUITING\"],\"orderBy\":\"LastUpdateSubmitDate:desc\",\"size\":25,\"offset\":0}":3,"health-study-condition:mucopolysaccharidosis-vii":23},{"pageToken":4,"total":5,"offset":6,"count":5,"results":7},null,4,0,[8,46,86,102],{"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":27,"overallStatus":33,"whyStopped":4,"lastUpdateSubmitDate":34,"lastUpdatePostDateStruct":35,"startDateStruct":38,"completionDateStruct":40,"leadSponsor":42,"locationsCount":45},"100332174","mucopolysaccharidosis-vii-disease-monitoring-program-100332174",false,"NCT03604835","Mucopolysaccharidosis VII Disease Monitoring Program","Mucopolysaccharidosis VII Disease Monitoring Program (MPS VII DMP)","Inclusion Criteria:\n\n* Diagnosis of MPS VII based on laboratory diagnosis, including either enzymatic or mutation analysis.\n* Willing and able to provide written informed consent or, in the case of patients under the age of 18 (or below adult ages as defined by local laws and regulations) or patients \\>18 years of age who have cognitive deficiencies, provide written assent (if required) and written informed consent by a legally authorized representative after the nature of the DMP has been explained, and prior to any research-related procedures.\n* Willing to comply with DMP visit schedule.\n\nExclusion Criteria:\n\n* Concurrent participation in other pharmaceutical company-sponsored interventional clinical trial unless approved by Ultragenyx.","ALL",{"count":18,"type":19},50,"ESTIMATED","OBSERVATIONAL","The objectives of this study are to characterize MPS VII disease presentation and progression and assess long-term effectiveness and safety, including hypersensitivity reactions and immunogenicity of vestronidase alfa.",[23,24,25,26],"Mucopolysaccharidosis VII","MPS VII","MPS 7","Sly Syndrome",[28,29,30,31,32],"UX003","Mepsevii","vestronidase alfa-vjbk","vestronidase alfa","recombinant human beta-glucuronidase","RECRUITING","2026-06-10",{"date":36,"type":37},"2026-06-11","ACTUAL",{"date":39,"type":37},"2018-01-29",{"date":41,"type":19},"2032-04",{"name":43,"class":44},"Ultragenyx Pharmaceutical Inc","INDUSTRY",14,{"id":47,"slug":48,"hasResults":11,"nctId":49,"briefTitle":50,"officialTitle":50,"acronym":51,"eligibilityCriteria":52,"healthyVolunteers":11,"sex":16,"minAge":4,"maxAge":53,"enrollmentInfo":54,"targetDuration":56,"studyType":20,"phases":4,"briefSummary":57,"conditions":58,"keywords":66,"overallStatus":33,"whyStopped":4,"lastUpdateSubmitDate":75,"lastUpdatePostDateStruct":76,"startDateStruct":78,"completionDateStruct":80,"leadSponsor":82,"locationsCount":85},"100486886","registry-of-patients-diagnosed-with-lysosomal-storage-diseases-100486886","NCT05619900","Registry of Patients Diagnosed With Lysosomal Storage Diseases","LSD Registry","Inclusion Criteria:\n\n* Patients aged 0-64 with a diagnosis of a lysosomal storage disease\n* Pregnant patients whose fetus has a diagnosis of a lysosomal storage disease\n\nExclusion Criteria:\n\n* There are no current exclusion criteria","64 Years",{"count":55,"type":19},250,"15 Years","This is an international prospective and retrospective registry of patients with Lysosomal Storage Diseases (LSDs) to understand the natural history of the disease and the outcomes of fetal therapies, with the overall goal of improving the prenatal management of patients with LSDs.",[59,60,61,62,23,63,64,65],"Mucopolysaccharidosis I","Mucopolysaccharidosis II","Mucopolysaccharidosis IV A","Mucopolysaccharidosis VI","Pompe Disease Infantile-Onset","Neuronopathic Gaucher Disease","Wolman Disease",[67,68,69,70,26,71,59,60,72,62,23,63,64,65,73,74],"Lysosomal Storage Disease","LSDs","Inborn Error of Metabolism","Hurler Syndrome","Hunter Syndrome","Mucopolysaccharidosis IVa","MPS","Mucopolysaccharidosis","2026-04-06",{"date":77,"type":37},"2026-04-08",{"date":79,"type":37},"2022-05-31",{"date":81,"type":19},"2050-05-31",{"name":83,"class":84},"University of California, San Francisco","OTHER",1,{"id":87,"slug":88,"hasResults":11,"nctId":89,"briefTitle":90,"officialTitle":4,"acronym":4,"eligibilityCriteria":91,"healthyVolunteers":11,"sex":16,"minAge":4,"maxAge":4,"enrollmentInfo":4,"targetDuration":4,"studyType":92,"phases":4,"briefSummary":93,"conditions":94,"keywords":95,"overallStatus":98,"whyStopped":4,"lastUpdateSubmitDate":75,"lastUpdatePostDateStruct":99,"startDateStruct":4,"completionDateStruct":4,"leadSponsor":101,"locationsCount":4},"100345244","expanded-access-to-mepsevii-100345244","NCT03775174","Expanded Access to Mepsevii","Inclusion Criteria:\n\n\\-\n\nExclusion Criteria:\n\n\\-","EXPANDED_ACCESS","Individual patient expanded access requests may be considered for patients who have no other treatment options",[24,23,26],[96,97],"Expanded Access","Compassionate Use","AVAILABLE",{"date":100,"type":37},"2026-04-07",{"name":43,"class":44},{"id":103,"slug":104,"hasResults":11,"nctId":105,"briefTitle":106,"officialTitle":107,"acronym":4,"eligibilityCriteria":108,"healthyVolunteers":11,"sex":16,"minAge":4,"maxAge":4,"enrollmentInfo":109,"targetDuration":4,"studyType":20,"phases":4,"briefSummary":111,"conditions":112,"keywords":4,"overallStatus":33,"whyStopped":4,"lastUpdateSubmitDate":117,"lastUpdatePostDateStruct":118,"startDateStruct":120,"completionDateStruct":122,"leadSponsor":124,"locationsCount":127},"100518911","mps-radico-cohort-radico-mps-100518911","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.",{"count":110,"type":19},1000,"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.",[59,60,113,114,62,23,115,116],"Mucopolysaccharidosis III","Mucopolysaccharidosis IV","Mucopolysaccharidosis IX","Multiple Sulfatase Deficiency Disease","2026-02-10",{"date":119,"type":37},"2026-02-11",{"date":121,"type":37},"2017-12-20",{"date":123,"type":19},"2026-12",{"name":125,"class":126},"Institut National de la Santé Et de la Recherche Médicale, France","OTHER_GOV",23]