Clinical Trial Update: Gene Therapy in Patients With Mucopolysaccharidosis Disease [Phase 1, Phase 2]

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Summary

This clinical trial update reports completion of a Phase 1/2 gene therapy study in patients with Mucopolysaccharidosis Type VI. The status on ClinicalTrials.gov…

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Diseases / indications

• Mucopolysaccharidosis Type Vi

Snippet

Sponsor: Fondazione Telethon | Conditions: Mucopolysaccharidosis Type VI | Phase 1, Phase 2 | Status: COMPLETED