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Browse Studies — Arctuva | Arctuva

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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

~$10 – $8,750

Ultragenyx Pharmaceutical IncNCT02716246

A Global, Open-Label, Phase I/II Trial, to Evaluate Safety, Tolerability, Pharmacodynamic and Preliminary Efficacy of JR-446 in Mucopolysaccharidosis Type IIIB (MPS IIIB)

This is a global, open-label, Phase I/II, interventional trial in participants younger than 6 years of age with Mucopolysaccharidosis Type IIIB (MPS IIIB), des…

Mucopolysaccharidosis IIIB

~$10 – $8,750

JCR Pharmaceuticals Co., Ltd.NCT07640984

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

~$10 – $6,250

Lundquist Institute for Biomedical Innovation at Harbor-UCLA Medical CenterNCT06333041