Why the study?
Important recent advances have been made in treating genetically caused neuromuscular diseases using therapies aimed at gene regulation, with several gaining regulatory approval or nearing approval.
Gene-based therapies, including ASOs, RNAi, and gene replacement, represent a major therapeutic revolution for monogenic neuromuscular disorders, offering disease-modifying treatments where none previously existed.
Gene regulation therapies advance options for genetic NMD; leaves open questions on long-term outcomes and equitable access.
Neuromuscular diseases (NMD) include a broad group of medical conditions with both acquired and genetic causes. In recent years, important advances have been made in the treatment of genetically caused NMD, and most of these advances are due to the implementation of therapies aimed at gene regulation. Among these therapies, gene replacement, small interfering RNA (siRNA), and antisense antinucleotides are the most promising approaches. More importantly, some of these therapies have already gained regulatory approval or are in the final stages of approval. The review focuses on motor neuron diseases, neuropathies, and Duchenne muscular dystrophy, summarizing the most recent developments in gene-based therapies for these conditions.
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Zanoteli et al. (2024) studied this question.
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