This update demonstrates the efficacy and safety of new treatments in Duchenne muscular dystrophy, suggesting a need for lasting evidence.
Recent therapeutic expansion has been driven in part by accelerated regulatory pathways relying on surrogate endpoints. While these advances provide important access, long-term efficacy, and safety data remain limited. Future progress will likely require rigorous clinical outcomes and rational combination of multimodal treatment strategies.
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Wilson et al. (2026) studied this question.
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