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NCT07554924

A Multicenter, Open-label, Dose-escalation Phase I/II Clinical Study to Evaluate the Safety, Preliminary Efficacy and Immunogenicity of SKG0201 Injection in Subjects With Spinal Muscular Atrophy Type I

Study Summary

This is a phase I/II clinical study to evaluate the safety, preliminary efficacy and immunogenicity of SKG0201 injection in subjects with Spinal Muscular Atrophy Type I.

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Interventions

SKG0201 InjectionGENETIC
SKG0201 is a recombinant adeno-associated virus (rAAV) vector-based gene therapy product.

Study Locations

FacilityCityStateCountry
Peking University First HospitalBeijingBeijing MunicipalityChina
Children's Hospital of Fudan UniversityShanghaiShanghai MunicipalityChina
Xinhua Hospital, Shanghai Jiao Tong University School of MedicineShanghaiShanghai MunicipalityChina
Children's Hospital, Zhejiang University School of MedicineHangzhouZhejiangChina

Official Trial Information

View on ClinicalTrials.gov

Data sourced from ClinicalTrials.gov. Last updated: May 29, 2026