Clinical Trial Update: Phase I/II/III Gene Transfer Clinical Trial of scAAV9.U1a.hSGSH [Phase 2, Phase 3]

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Summary

Ultragenyx is conducting a Phase 2/3 gene therapy trial (scAAV9.U1a.hSGSH) for Mucopolysaccharidosis Type IIIA (Sanfilippo syndrome type A); the study is curren…

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Diseases / indications

• Mucopolysaccharidosis Type Iiia
• Sanfilippo Syndrome Type

Drugs mentioned

• scAAV9.U1a.hSGSH

Snippet

Sponsor: Ultragenyx Pharmaceutical Inc | Conditions: MPS IIIA, Sanfilippo Syndrome | Phase 2, Phase 3 | Status: RECRUITING