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NCT06392724

A Single-arm, Open-label, Single-center Study to Evaluate the Safety and Tolerability of Intravenous GEN6050X Gene Therapy in Ambulatory Boys With Duchenne Muscular Dystrophy (DMD).

Study Summary

The study will evaluate the safety and tolerability of GEN6050X gene therapy in Duchenne muscular dystrophy (DMD) patients amenable to exon 50 skipping.

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Interventions

GEN6050X intravenous injectionGENETIC
GEN6050X is an intravenously administered human DMD exon 50 skipping base editing drug.

Study Locations

FacilityCityStateCountry
Peking Union Medical College HospitalBeijingChina

Official Trial Information

View on ClinicalTrials.gov

Data sourced from ClinicalTrials.gov. Last updated: April 14, 2026