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drugset / Trial / NCT06191354

A Clinical Study Evaluating the Safety and Efficacy of SKG0201 Injection in Patients With Spinal Muscular Atrophy Type 1

NCT06191354 ↗

Active not recruiting 12 enrolled Kun Sun
NaSingle-groupOpen-labelTreatment

Summary

This is a clinical study to evaluate the safety and efficacy of gene therapy drug SKG0201 Injection in patients with spinal muscular atrophy Type 1 (SMA 1).

Timeline

Start
2023-06-25
Primary completion
2025-12
Completion
2025-12

Drugs

EvaluationDrugModalityDoseRoute
Subject SKG0201 Gene therapy (AAV / viral vector) — —

Indications

spinal muscular atrophy, type 1