{"status":"ok","message-type":"work","message-version":"1.0.0","message":{"indexed":{"date-parts":[[2026,9,28]],"date-time":"2026-09-28T19:09:33Z","timestamp":1790622573236,"version":"4.1.0"},"reference-count":34,"publisher":"American Society for Clinical Investigation","issue":"21","content-domain":{"domain":[],"crossmark-restriction":false},"short-container-title":[],"published-print":{"date-parts":[[2016,12,22]]},"abstract":"<jats:p>\n                    Duchenne muscular dystrophy (DMD) is a devastating muscle disease characterized by progressive muscle deterioration and replacement with an aberrant fatty, fibrous matrix. Chronic upregulation of nuclear factor \u03baB (NF-\u03baB) is implicated as a driver of the dystrophic pathogenesis. Herein, 2 members of a novel class of NF-\u03baB inhibitors, edasalonexent (formerly CAT-1004) and CAT-1041, were evaluated in both\n                    <jats:italic>mdx<\/jats:italic>\n                    mouse and golden retriever muscular dystrophy (GRMD) dog models of DMD. These orally bioavailable compounds consist of a polyunsaturated fatty acid conjugated to salicylic acid and potently suppress the pathogenic NF-\u03baB subunit p65\/RelA in vitro. In vivo, CAT-1041 effectively improved the phenotype of\n                    <jats:italic>mdx<\/jats:italic>\n                    mice undergoing voluntary wheel running, in terms of activity, muscle mass and function, damage, inflammation, fibrosis, and cardiac pathology. We identified significant increases in dysferlin as a possible contributor to the protective effect of CAT-1041 to sarcolemmal damage. Furthermore, CAT-1041 improved the more severe GRMD phenotype in a canine case study, where muscle mass and diaphragm function were maintained in a treated GRMD dog. These results demonstrate that NF-\u03baB modulation by edasalonexent and CAT-1041 is effective in ameliorating the dystrophic process and these compounds are candidates for new treatments for DMD patients.\n                  <\/jats:p>","DOI":"10.1172\/jci.insight.90341","type":"journal-article","created":{"date-parts":[[2016,12,21]],"date-time":"2016-12-21T11:08:39Z","timestamp":1482318519000},"source":"Crossref","is-referenced-by-count":48,"title":["Disease-modifying effects of orally bioavailable NF-\u03baB inhibitors in dystrophin-deficient muscle"],"prefix":"10.1172","volume":"1","author":[{"given":"David W.","family":"Hammers","sequence":"first","affiliation":[{"name":"Department of Physiology and"},{"name":"Pennsylvania Muscle Institute, University of Pennsylvania Perelman School of Medicine, Philadelphia, Pennsylvania, USA."},{"name":"Department of Pharmacology and Therapeutics,"},{"name":"Myology Institute and"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Margaret M.","family":"Sleeper","sequence":"additional","affiliation":[{"name":"Myology Institute and"},{"name":"Department of Clinical Studies, University of Pennsylvania School of Veterinary Medicine, Philadelphia, Pennsylvania, USA."},{"name":"Department of Small Animal Clinical Sciences, University of Florida College of Veterinary Medicine,"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Sean C.","family":"Forbes","sequence":"additional","affiliation":[{"name":"Myology Institute and"},{"name":"Department of Physical Therapy, University of Florida, Gainesville, Florida, USA."}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Cora C.","family":"Coker","sequence":"additional","affiliation":[{"name":"Department of Pharmacology and Therapeutics,"},{"name":"Myology Institute and"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Michael R.","family":"Jirousek","sequence":"additional","affiliation":[{"name":"Catabasis Pharmaceuticals, Inc., Cambridge, Massachusetts, USA."}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Michael","family":"Zimmer","sequence":"additional","affiliation":[{"name":"Catabasis Pharmaceuticals, Inc., Cambridge, Massachusetts, USA."}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Glenn A.","family":"Walter","sequence":"additional","affiliation":[{"name":"Myology Institute and"},{"name":"Department of Physiology and Functional Genomics, University of Florida College of Medicine, Gainesville, Florida, USA."}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"H. Lee","family":"Sweeney","sequence":"additional","affiliation":[{"name":"Department of Physiology and"},{"name":"Department of Pharmacology and Therapeutics,"},{"name":"Myology Institute 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