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Sanfilippo Syndrome A
Clinical trial pipeline · Data from ClinicalTrials.gov
See which Sanfilippo Syndrome A trials you may qualify forClinical trial pipeline · Data from ClinicalTrials.gov
See which Sanfilippo Syndrome A trials you may qualify forThe main objective of this study is to evaluate the efficacy and safety of UX111 for the treatment of MPS IIIA.
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 primary objective of the study is to determine the safety and feasibility of intrathecal administration of DUOC-01 as an adjunctive therapy in patients with…
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).
This study is planned to document, through retrospective and prospective data collection, syndrome progression in children and young adults with MPS IIIC.
The main objective of this study is to evaluate the safety/tolerability and efficacy of UX111 (previously known as ABO-102) in participants with Mucopolysacchar…
This is a multicenter, open-label, Phase 1/2 study to assess the safety, tolerability, pharmacokinetics (PK), pharmacodynamics (PD) and clinical efficacy of DNL…