Meeting report discusses advancements in gene therapy for neuromuscular disorders, highlighting challenges and regulatory pathways.
The 2025 MDA Gene Therapy Summit brought together leading researchers, clinicians, and industry representatives to discuss the latest advancements and challenges in the development and clinical translation of genetic therapies for neuromuscular disorders. The meeting featured in-depth presentations on clinical trials, therapeutic developments, and regulatory pathways for AAV-mediated gene therapies, highlighting both successes and challenges in the field.
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Lin et al. (2026) studied this question.
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