[{"data":1,"prerenderedAt":-1},["ShallowReactive",2],{"health-studies-list:{\"conditionNormalized\":\"pgbm2\",\"overallStatus\":[\"RECRUITING\",\"AVAILABLE\",\"NOT_YET_RECRUITING\"],\"orderBy\":\"LastUpdateSubmitDate:desc\",\"size\":25,\"offset\":0}":3,"health-study-condition:pgbm2":56},{"pageToken":4,"total":5,"offset":6,"count":5,"results":7},null,1,0,[8],{"id":9,"slug":4,"hasResults":10,"nctId":11,"briefTitle":12,"officialTitle":12,"acronym":4,"eligibilityCriteria":13,"healthyVolunteers":10,"sex":14,"minAge":15,"maxAge":16,"enrollmentInfo":17,"targetDuration":4,"studyType":20,"phases":4,"briefSummary":21,"conditions":22,"keywords":38,"overallStatus":44,"whyStopped":4,"lastUpdateSubmitDate":45,"lastUpdatePostDateStruct":46,"startDateStruct":49,"completionDateStruct":51,"leadSponsor":53,"locationsCount":5},"100577196",false,"NCT06795152","Rare Glycogen Storage Diseases Natural History Study","Inclusion Criteria:\n\n* Diagnosis of a rare GSD, including 0a, 0b, VII, X, XII, XIII, XV, PRKAG2 syndrome or Danon disease\n\n  * Two variants in the gene associated with the specific GSD type (for autosomal recessive diseases)\n  * One variant in the gene associated with the specific GSD type (for autosomal dominant or X-linked diseases)\n  * Deficient enzyme activity in liver, muscle, skin fibroblast or other tissue\n  * One variant in causative gene with evidence of disease, per a clinician\n  * Histology as confirmed by a clinician\n* Able to provide informed consent for self (adults) or affected individual (minor or adults with a legally authorized representative)\n* Able to provide consent for release of medical records\n* Pregnant women with a diagnosis of a rare GSD will be included\n\nExclusion Criteria:\n\n* Unable to provide informed consent for participation for one's self or by legally authorized representative\u002Flegal guardian\u002Fparent","ALL","0 Years","90 Years",{"count":18,"type":19},200,"ESTIMATED","OBSERVATIONAL","The purpose of this study is to collect and study key medical data about several ultra-rare GSDs (Glycogen Storage Diseases) including, but not limited to, GSD types 0a, 0b, VII, X, XII, XIII, XV, PRKAG2 syndrome and Danon disease.",[23,24,25,26,27,28,29,30,31,32,33,34,35,36,37],"Glycogen Storage Disease","GSD Type 0A","GSD Type 0B","GSD VII","Tarui Disease","GSD X","GSD XII","GSD XIII","GSD XV","PGBM2","PRKAG2","Danon Disease","Polyglucosan Body Myopathy Type 1","Polyglucosan Body Myopathy Type 2","RBCK1 Deficiency",[39,40,41,36,32,33,42,35,43],"glycogen storage disease","GSD","Tarui disease","Danon disease","RBCK1","RECRUITING","2026-01-05",{"date":47,"type":48},"2026-01-07","ACTUAL",{"date":50,"type":48},"2024-12-23",{"date":52,"type":19},"2034-12",{"name":54,"class":55},"Duke University","OTHER",""]