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Mucopolysaccharidosis III-A
Clinical trial pipeline · Data from ClinicalTrials.gov
See which Mucopolysaccharidosis III-A trials may be worth asking aboutClinical trial pipeline · Data from ClinicalTrials.gov
See which Mucopolysaccharidosis III-A trials may be worth asking aboutNorth America
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The main objective of this study is to evaluate the efficacy and safety of UX111 for the treatment of MPS IIIA.
This study is planned to document, through retrospective and prospective data collection, syndrome progression in children and young adults with MPS IIIC.
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…
The purpose of this study is to evaluate the safety, tolerability, efficacy, pharmacokinetics and pharmacodynamics of recombinant human heparan N-sulfatase (rhH…
A dose escalation study to evaluate the safety, tolerability, and pharmacologic properties of Ambroxol in adult participants with Sanfilippo disease(s) (MPS3).
A Global Phase III multicenter, randomized, assessor-blinded, active-controlled designed to evaluate safety and efficacy of study drug for the treatment of the…
The primary objectives of this study are to evaluate the effects of Tralesinidase Alfa (TA) on cognition