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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

An Open-Label, Single Injection, Ascending Dose Phase I/II Clinical Study of rAAV9-CMV-hNAGLUop Gene for Patients With Mucopolysaccharidosis (MPS) IIIB

The goal of this clinical trial is to learn if one infusion of rAAV9-CMV-hNAGLUop can treat patients with Sanfilippo syndrome (MPS III) 6 months or older. The …

Sanfilippo Syndrome (MPS III)

~$10 – $7,500

NeuroGTNCT07818759

Longitudinal Study of Neurodegenerative Disorders

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 understandi…

MLDKrabbe DiseaseALD+24 more

~$10 – $6,250

University of PittsburghNCT03333200