{"status":"ok","message-type":"work","message-version":"1.0.0","message":{"indexed":{"date-parts":[[2026,9,21]],"date-time":"2026-09-21T00:12:18Z","timestamp":1789949538904,"version":"4.0.1"},"reference-count":87,"publisher":"Wiley","issue":"11-12","license":[{"start":{"date-parts":[[2013,12,27]],"date-time":"2013-12-27T00:00:00Z","timestamp":1388102400000},"content-version":"vor","delay-in-days":56,"URL":"http:\/\/onlinelibrary.wiley.com\/termsAndConditions#vor"}],"content-domain":{"domain":[],"crossmark-restriction":false},"short-container-title":["The Journal of Gene Medicine"],"published-print":{"date-parts":[[2013,11]]},"abstract":"<jats:title>Abstract<\/jats:title><jats:p>This review updates the state\u2010of\u2010the art accomplishments of the multifaceted gene\u2010based therapies, which include DNA or RNA as either therapeutic tools or targets for the treatment of neuromuscular diseases. It also provides insights into the key role that patient organizations have played in research and development; in particular, by addressing bottlenecks and generating boundary conditions that have contributed to scientific breakthroughs, and the effectiveness of innovation processes. Several gene therapy methods have reached the clinical stage and are now addressing both specific and classical issues related to this novel technology. Not ready yet for clinical application, genome editing is at its infancy. More rapidly progressing, RNA\u2010based therapeutics, and especially exon skipping, exon inclusion and stop codon readthrough strategies, are about to move to the market. Most importantly, patients were at the forefront of this discovery process, from basic knowledge to innovation and translational research in a rapidly growing field of unmet medical needs. In recent years, Duchenne muscular dystrophy was the fertile ground for new therapeutic concepts that have been extended to other neuromuscular disorders, such as spinal muscular atrophy, myotonic dystrophies or fascioscapulohumeral dystrophy. In line with their longstanding policy, patient organizations will keep working in a proactive manner to bring together all stakeholders with a view to working out truly therapeutic solutions over a long\u2010term perspective. Copyright \u00a9 2013 John Wiley &amp; Sons, Ltd.<\/jats:p>","DOI":"10.1002\/jgm.2747","type":"journal-article","created":{"date-parts":[[2013,10,12]],"date-time":"2013-10-12T02:32:33Z","timestamp":1381545153000},"page":"397-413","source":"Crossref","is-referenced-by-count":14,"title":["Gene\u2010based therapies of neuromuscular disorders: an update and the pivotal role of patient organizations in their discovery and implementation"],"prefix":"10.1002","volume":"15","author":[{"given":"Serge","family":"Braun","sequence":"first","affiliation":[{"name":"AFM\u2010Telethon  Evry Cedex France"}],"role":[{"vocabulary":"crossref","role":"author"}]}],"member":"311","published-online":{"date-parts":[[2013,12,27]]},"reference":[{"key":"e_1_2_8_2_1","volume-title":"Proceedings of the Sixth International Congress of Genetics, Vol. 1. Transactions and General Addresses, Vol. 2. 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