Clinical Trial: Natural History to Assess Disease in Patients With MPS IIIC

U.S., July 18 -- ClinicalTrials.gov registry received information related to the study (NCT07712003) titled 'Natural History to Assess Disease in Patients With MPS IIIC' on June 15.

Brief Summary: This is a REMOTE prospective observational study of participants with MPS IIIC.

No investigational medicinal product will be administered as part of this study.

Patients functional abilities will be captured using video recordings taken by patients legal representative or caregiver using a dedicated clinical video application downloaded from the internet onto a smartphone. The video application is called C-RARE.

Electronic observer-reported outcome (ObsRO) questionnaires will be completed by the legal representatives or caregivers via C-RARE....