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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

Ultragenyx Pharmaceutical IncNCT02716246

Arctuva

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