Open access
Aug 2026
Gene therapy for rare diseases marks a new era in precision medicine: Insights from clinical trials
Clinical evidence suggests that gene-based therapies in the management of rare diseases can achieve sustained functional benefits, reduce disease-related complications, and lessen dependence on long-term replacement or supportive treatments.
S. Suprianto, Y. Messe, Raehan AH. Hamzah et al.
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