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Observational Study to Assess Disease Symptoms and Progression in Participants With Sanfilippo Syndrome Type C (Mucopolysaccharidosis Type III [MPS III] C)
This is a REMOTE prospective observational study of participants with MPS IIIC. No investigational medicinal product will be administered as part of this stud…
Sanfilippo Syndrome Type CMucopolysaccharidosis (MPS) IIIC
Estimated
~$10 – $1,125
Phoenix NestNCT07712003
A Combination Retrospective and Prospective Natural History Study of Participants With Sanfilippo Syndrome Mucopolysaccharidosis Type IIIC (MPS IIIC)
This study is planned to document, through retrospective and prospective data collection, syndrome progression in children and young adults with MPS IIIC.
Sanfilippo Syndrome Type C
Estimated
~$10 – $1,125
Phoenix NestNCT05825131