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With the caveat that many products still fail to translate the positive results obtained in pre-clinical models to humans, both the technological development (e.g., implementation of tissue-specific vectors) as well as advances on the knowledge of pathogenetic mechanisms form a collective foundation for potentially curative approaches to these debilitating conditions. This review delineates the current panorama of therapies targeting the most prevalent forms of inherited neuromuscular diseases, emphasizing approved treatments and those already undergoing human testing, offering insights into the state-of-the-art interventions.<\/jats:p>","DOI":"10.1007\/s00018-024-05229-9","type":"journal-article","created":{"date-parts":[[2024,4,28]],"date-time":"2024-04-28T08:01:31Z","timestamp":1714291291000},"update-policy":"https:\/\/doi.org\/10.1007\/springer_crossmark_policy","source":"Crossref","is-referenced-by-count":20,"title":["Molecular mechanisms and therapeutic strategies for neuromuscular diseases"],"prefix":"10.1007","volume":"81","author":[{"given":"Alberto Andrea","family":"Zambon","sequence":"first","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Yuri Matteo","family":"Falzone","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Alessandra","family":"Bolino","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"ORCID":"https:\/\/orcid.org\/0000-0003-2546-4357","authenticated-orcid":false,"given":"Stefano Carlo","family":"Previtali","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]}],"member":"297","published-online":{"date-parts":[[2024,4,28]]},"reference":[{"key":"5229_CR1","doi-asserted-by":"publisher","DOI":"10.1126\/scitranslmed.abb6871","author":"L Kong","year":"2021","unstructured":"Kong L, Valdivia DO, Simon CM et al (2021) Impaired prenatal motor axon development necessitates early therapeutic intervention in severe SMA. 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