Back to search
Sanfilippo Syndrome
Clinical trial pipeline · Data from ClinicalTrials.gov
See which Sanfilippo Syndrome trials you may qualify forClinical trial pipeline · Data from ClinicalTrials.gov
See which Sanfilippo Syndrome 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 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…
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…
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…
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 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…