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Showing 1-1 of 1 trials for Mucopolysaccharidosis-iii
Recruiting

Phase I/II/III Gene Transfer Clinical Trial of scAAV9.U1a.hSGSH

Ohio · Columbus, OH

The main objective of this study is to evaluate the efficacy and safety of UX111 for the treatment of MPS IIIA.

MPS IIIASanfilippo SyndromeSanfilippo AMucopolysaccharidosis III
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