RBCK1 Deficiency

1

Review clinical trials related to RBCK1 Deficiency. Use filters to narrow results by trial status, phase, treatment, biological sex and sponsor.

Condition / disease
Location
Status: Recruiting

Rare Glycogen Storage Diseases Natural History Study

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.

Participants needed: 200
Trial details
Age: 0-90Biological sex: AllType: ObservationalSponsor: Duke UniversityUpdated: Jan 7, 2026Locations: 1
Eligibility criteria

Diagnosis of a rare GSD, including 0a, 0b, VII, X, XII, XIII, XV, PRKAG2 syndrom... [+8]

Unable to provide informed consent for participation for one's self or by legall...