Clinical Trial Update: SKG0201 Gene Therapy for Spinal Muscular Atrophy Type 1 (SMA 1)
Trial ID: NCT06191354Study Title: A Multicenter, Open, Dose-Escalation Clinical Study Evaluating the Safety, Init...
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What is gene therapy?
Gene therapy acts as the lighthouse of contemporary medicine by offering to help correct hereditary afflictions at their very roots—the genome itself. Such treatment involves ...
rare-disease-treatment
rare-disease-treatment