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Phase 2
Phase I/II/III Gene Transfer Clinical Trial of scAAV9.U1a.hSGSH for Mucopolysaccharidosis (MPS) IIIA
The main objective of this study is to evaluate the efficacy and safety of UX111 for the treatment of MPS IIIA.
MPS IIIASanfilippo SyndromeSanfilippo A+1 more
Ultragenyx Pharmaceutical IncNCT02716246
Phase 2
Randomized, Double-Blind, Placebo-Controlled Cross-Over Study to Evaluate the Effects of Cannabidiol on Neurobehavioral and Function Outcomes in Sanfilippo Syndrome
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…
Sanfilippo SyndromeMucopolysaccharidosis III
Lundquist Institute for Biomedical Innovation at Harbor-UCLA Medical CenterNCT06333041