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FSHD is the result of a toxic gain-of-function from de-repression of the DUX4 gene, a gene not normally expressed in skeletal muscle. With a clear therapeutic target, there is increasing interest in drug development for FSHD, an interest buoyed by the recent therapeutic successes in other neuromuscular diseases. Herein, we review the underlying disease mechanism, potential therapeutic approaches as well as the state of trial readiness in the planning and execution of future clinical trials in FSHD.<\/jats:p>","DOI":"10.3233\/jnd-200554","type":"journal-article","created":{"date-parts":[[2020,11,17]],"date-time":"2020-11-17T12:38:18Z","timestamp":1605616698000},"page":"441-451","update-policy":"https:\/\/doi.org\/10.1177\/sage-journals-update-policy","source":"Crossref","is-referenced-by-count":35,"title":["Current Therapeutic Approaches in FSHD"],"prefix":"10.1177","volume":"8","author":[{"given":"Leo H.","family":"Wang","sequence":"first","affiliation":[{"name":"Department of Neurology, University of Washington, Seattle, WA, USA"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Rabi","family":"Tawil","sequence":"additional","affiliation":[{"name":"Department of Neurology, University of Rochester, Rochester, NY, USA"}],"role":[{"vocabulary":"crossref","role":"author"}]}],"member":"179","published-online":{"date-parts":[[2020,11,10]]},"reference":[{"key":"ref001","doi-asserted-by":"publisher","DOI":"10.1212\/WNL0000000000000797"},{"key":"ref002","doi-asserted-by":"publisher","DOI":"10.1016\/s0960-8966(01)00201-2"},{"key":"ref003","doi-asserted-by":"publisher","DOI":"10.1016\/j.clineuro.2011.10.022"},{"key":"ref004","doi-asserted-by":"publisher","DOI":"10.1212\/wnl.0000000000001783"},{"key":"ref005","doi-asserted-by":"publisher","DOI":"10.1002\/mus.20522"},{"key":"ref006","doi-asserted-by":"publisher","DOI":"10.1212\/wnl.48.1.38"},{"key":"ref007","doi-asserted-by":"publisher","DOI":"10.1016\/j.nmd.2013.01.008"},{"key":"ref008","doi-asserted-by":"publisher","DOI":"10.1186\/1471-2164-9-533"},{"key":"ref009","doi-asserted-by":"publisher","DOI":"10.1002\/ana.25326"},{"key":"ref010","doi-asserted-by":"publisher","DOI":"10.1212\/WNL.0b013e3181f96175"},{"key":"ref011","doi-asserted-by":"publisher","DOI":"10.1093\/hmg\/ddu486"},{"key":"ref012","doi-asserted-by":"publisher","DOI":"10.1038\/ng.2454"},{"key":"ref013","doi-asserted-by":"publisher","DOI":"10.1038\/ng1262"},{"key":"ref014","doi-asserted-by":"publisher","DOI":"10.1016\/j.ajhg.2016.03.013"},{"key":"ref015","doi-asserted-by":"publisher","DOI":"10.1212\/WNL.0000000000009617"},{"key":"ref016","doi-asserted-by":"publisher","DOI":"10.1212\/WNL.0000000000007456"},{"key":"ref017","doi-asserted-by":"publisher","DOI":"10.1002\/humu.21091"},{"key":"ref018","doi-asserted-by":"publisher","DOI":"10.1038\/ng.3858"},{"key":"ref019","doi-asserted-by":"publisher","DOI":"10.1371\/journal.pgen.1001181"},{"key":"ref020","doi-asserted-by":"publisher","DOI":"10.1002\/dvdy.22210"},{"key":"ref021","doi-asserted-by":"publisher","DOI":"10.1016\/j.devcel.2011.11.013"},{"key":"ref022","doi-asserted-by":"publisher","DOI":"10.1371\/journal.pone.0160022"},{"key":"ref023","doi-asserted-by":"publisher","DOI":"10.1172\/jci.insight.123538"},{"key":"ref024","doi-asserted-by":"publisher","DOI":"10.1093\/hmg\/dds284"},{"key":"ref025","doi-asserted-by":"publisher","DOI":"10.1002\/ana.22275"},{"key":"ref026","doi-asserted-by":"publisher","DOI":"10.1016\/j.expneurol.2008.07.022"},{"key":"ref027","doi-asserted-by":"publisher","DOI":"10.1186\/s13072-018-0215-z"},{"key":"ref028","doi-asserted-by":"publisher","DOI":"10.1093\/hmg\/ddv315"},{"key":"ref029","doi-asserted-by":"publisher","DOI":"10.1242\/jcs.180372"},{"key":"ref030","doi-asserted-by":"publisher","DOI":"10.1073\/pnas.0708659104"},{"key":"ref031","doi-asserted-by":"publisher","DOI":"10.1038\/emboj.2008.201"},{"key":"ref032","doi-asserted-by":"publisher","DOI":"10.1016\/s0960-8966(02)00284-5"},{"key":"ref033","doi-asserted-by":"publisher","DOI":"10.1093\/hmg\/ddu251"},{"issue":"1","key":"ref034","first-page":"46","volume":"48","author":"Tawil R","year":"1997","journal-title":"FSH-DY Group. 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