Clinical Trial Update: Safety and Efficacy of AAV9. hMCOLN1co For Patients With Mucolipidosis Type IV [Phase 1]

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Summary

This Clinical Trial Update covers a Phase 1 study of AAV9.hMCOLN1co for patients with Mucolipidosis Type IV. The study is currently enrolling by invitation.

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Diseases / indications

• Mucolipidosis Type Iv

Drugs mentioned

• AAV9.hMCOLN1co

Snippet

Sponsor: The Children's Hospital of Zhejiang University School of Medicine | Conditions: Mucolipidosis Type IV | Phase 1 | Status: ENROLLING_BY_INVITATION