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Disclosures Dr McNally is or has been a consultant for Amgen, AstraZeneca, Avidity Biosciences, Cytokinetics, Pfizer, PepGen, and Tenaya Therapeutics and is the founder of Ikaika Therapeutics. The other authors report no conflicts.
Gloss D RTM III, Ashwal S, Oskoui M. Practice guideline update summary: Corticosteroid treatment of Duchenne muscular dystrophy. Report of the Guideline Development Subcommittee of the American Academy of Neurology. 2016;86:465–472. doi: 10.1212/wnl.0000000000002337
-
DOI
-
PMC
-
PubMed
Moxley RT 3rd, Pandya S, Ciafaloni E, Fox DJ, Campbell K. Change in natural history of Duchenne muscular dystrophy with long-term corticosteroid treatment: implications for management. J Child Neurol. 2010;25:1116–1129. doi: 10.1177/0883073810371004
-
DOI
-
PubMed
Buddhe S, Cripe L, Friedland-Little J, Kertesz N, Eghtesady P, Finder J, Hor K, Judge DP, Kinnett K, McNally EM, et al. Cardiac Management of the Patient With Duchenne Muscular Dystrophy. Pediatrics. 2018;142:S72–s81. doi: 10.1542/peds.2018-0333I
-
DOI
-
PMC
-
PubMed
Porcher R, Desguerre I, Amthor H, Chabrol B, Audic F, Rivier F, Isapof A, Tiffreau V, Campana-Salort E, Leturcq F, et al. Association between prophylactic angiotensin-converting enzyme inhibitors and overall survival in Duchenne muscular dystrophy-analysis of registry data. Eur Heart J. 2021;42:1976–1984. doi: 10.1093/eurheartj/ehab054
-
DOI
-
PubMed
Filonova G, Aartsma-Rus A. Next steps for the optimization of exon therapy for Duchenne muscular dystrophy. Expert Opin Biol Ther. 2023;23:133–143. doi: 10.1080/14712598.2023.2169070
-
DOI
-
PubMed