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Mucopolysaccharidosis III
Clinical trial pipeline · Data from ClinicalTrials.gov
See which Mucopolysaccharidosis III trials may be worth asking aboutClinical trial pipeline · Data from ClinicalTrials.gov
See which Mucopolysaccharidosis III trials may be worth asking aboutNorth America
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Trials may have sites in multiple countries. Filtering shows any trial with at least one site in the selected country.
The main objective of this study is to evaluate the efficacy and safety of UX111 for the treatment of MPS IIIA.
The goal of this clinical trial is to test cannabidiol in Sanfilippo syndrome. The main questions it aims to answer are: 1) determine the safety of cannabidiol…
This study is planned to document, through retrospective and prospective data collection, syndrome progression in children and young adults with MPS IIIC.
The goal of this observational study is to characterize the epidemiology and natural history of MPS diseases by building a retrospective and prospective collect…
The purpose of this study is to understand the course of rare genetic disorders that affect the brain. This data is being analyzed to gain a better understandin…
A Phase I/ II, open-label study, designed to evaluate the safety and explore efficacy of the study drug in development for the treatment of MPS IIIB patients.
The primary objectives of this study are to evaluate the effects of Tralesinidase Alfa (TA) on cognition
This is an intermediate-size patient population Early Access Program (EAP) providing access to intracerebroventricular (ICV) tralesinidase alfa (TA) for partici…