Clinical Trial Update: Single-Dose AAV-MECP2 Safety/Tolerability and Efficacy in Rett Syndrome [EARLY_Phase 1]

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Summary

A single-dose AAV-MECP2 gene therapy trial in Rett syndrome is in early Phase 1 and currently recruiting to assess safety, tolerability, and preliminary efficac…

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Diseases / indications

• Rett Syndrome

Drugs mentioned

• AAV-MECP2 (AAV-MECP2 gene therapy)

Snippet

Sponsor: Guangzhou Women and Children's Medical Center | Conditions: Rett Syndrome | EARLY_Phase 1 | Status: RECRUITING