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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)

Estimated

~$150 – $4,500

Actual pay is set by the research site.

NeuroGTNCT07818759