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Pharmacotherapy of Duchenne Muscular Dystrophy.

Hoffman EP.

Handb Exp Pharmacol. 2019 Aug 3. doi: 10.1007/164_2019_256. [Epub ahead of print]


Corticosteroids for the treatment of Duchenne muscular dystrophy.

Matthews E, Brassington R, Kuntzer T, Jichi F, Manzur AY.

Cochrane Database Syst Rev. 2016 May 5;(5):CD003725. doi: 10.1002/14651858.CD003725.pub4. Review.


Restoring Dystrophin Expression in Duchenne Muscular Dystrophy: Current Status of Therapeutic Approaches.

Shimizu-Motohashi Y, Komaki H, Motohashi N, Takeda S, Yokota T, Aoki Y.

J Pers Med. 2019 Jan 7;9(1). pii: E1. doi: 10.3390/jpm9010001. Review.


Dystrophin quantification and clinical correlations in Becker muscular dystrophy: implications for clinical trials.

Anthony K, Cirak S, Torelli S, Tasca G, Feng L, Arechavala-Gomeza V, Armaroli A, Guglieri M, Straathof CS, Verschuuren JJ, Aartsma-Rus A, Helderman-van den Enden P, Bushby K, Straub V, Sewry C, Ferlini A, Ricci E, Morgan JE, Muntoni F.

Brain. 2011 Dec;134(Pt 12):3547-59. doi: 10.1093/brain/awr291. Epub 2011 Nov 18. Erratum in: Brain. 2016 Apr;139(Pt 4):e27.


Quantitative Antisense Screening and Optimization for Exon 51 Skipping in Duchenne Muscular Dystrophy.

Echigoya Y, Lim KRQ, Trieu N, Bao B, Miskew Nichols B, Vila MC, Novak JS, Hara Y, Lee J, Touznik A, Mamchaoui K, Aoki Y, Takeda S, Nagaraju K, Mouly V, Maruyama R, Duddy W, Yokota T.

Mol Ther. 2017 Nov 1;25(11):2561-2572. doi: 10.1016/j.ymthe.2017.07.014. Epub 2017 Jul 28.


Contributions of Japanese patients to development of antisense therapy for DMD.

Matsuo M, Takeshima Y, Nishio H.

Brain Dev. 2016 Jan;38(1):4-9. doi: 10.1016/j.braindev.2015.05.014. Epub 2015 Jun 18. Review.


Biochemical characterization of patients with in-frame or out-of-frame DMD deletions pertinent to exon 44 or 45 skipping.

Anthony K, Arechavala-Gomeza V, Ricotti V, Torelli S, Feng L, Janghra N, Tasca G, Guglieri M, Barresi R, Armaroli A, Ferlini A, Bushby K, Straub V, Ricci E, Sewry C, Morgan J, Muntoni F.

JAMA Neurol. 2014 Jan;71(1):32-40. doi: 10.1001/jamaneurol.2013.4908.


An Overview of Recent Advances and Clinical Applications of Exon Skipping and Splice Modulation for Muscular Dystrophy and Various Genetic Diseases.

Rodrigues M, Yokota T.

Methods Mol Biol. 2018;1828:31-55. doi: 10.1007/978-1-4939-8651-4_2. Review.


Mutation-Based Therapeutic Strategies for Duchenne Muscular Dystrophy: From Genetic Diagnosis to Therapy.

Nakamura A.

J Pers Med. 2019 Mar 4;9(1). pii: E16. doi: 10.3390/jpm9010016. Review.


Exon Skipping Therapy Using Phosphorodiamidate Morpholino Oligomers in the mdx52 Mouse Model of Duchenne Muscular Dystrophy.

Miyatake S, Mizobe Y, Takizawa H, Hara Y, Yokota T, Takeda S, Aoki Y.

Methods Mol Biol. 2018;1687:123-141. doi: 10.1007/978-1-4939-7374-3_9.


In Vitro Multiexon Skipping by Antisense PMOs in Dystrophic Dog and Exon 7-Deleted DMD Patient.

Nakamura A, Aoki Y, Tsoumpra M, Yokota T, Takeda S.

Methods Mol Biol. 2018;1828:151-163. doi: 10.1007/978-1-4939-8651-4_9.


Anti-dystrophin T cell responses in Duchenne muscular dystrophy: prevalence and a glucocorticoid treatment effect.

Flanigan KM, Campbell K, Viollet L, Wang W, Gomez AM, Walker CM, Mendell JR.

Hum Gene Ther. 2013 Sep;24(9):797-806. doi: 10.1089/hum.2013.092.


Multiple Exon Skipping in the Duchenne Muscular Dystrophy Hot Spots: Prospects and Challenges.

Echigoya Y, Lim KRQ, Nakamura A, Yokota T.

J Pers Med. 2018 Dec 7;8(4). pii: E41. doi: 10.3390/jpm8040041. Review.


[Genetic Diagnosis and Molecular Therapies for Duchenne Muscular Dystrophy].

Takeshima Y.

Rinsho Byori. 2015 Oct;63(10):1194-201. Review. Japanese.


NS-065/NCNP-01: An Antisense Oligonucleotide for Potential Treatment of Exon 53 Skipping in Duchenne Muscular Dystrophy.

Watanabe N, Nagata T, Satou Y, Masuda S, Saito T, Kitagawa H, Komaki H, Takagaki K, Takeda S.

Mol Ther Nucleic Acids. 2018 Dec 7;13:442-449. doi: 10.1016/j.omtn.2018.09.017. Epub 2018 Sep 27.


A duchenne muscular dystrophy gene hot spot mutation in dystrophin-deficient cavalier king charles spaniels is amenable to exon 51 skipping.

Walmsley GL, Arechavala-Gomeza V, Fernandez-Fuente M, Burke MM, Nagel N, Holder A, Stanley R, Chandler K, Marks SL, Muntoni F, Shelton GD, Piercy RJ.

PLoS One. 2010 Jan 13;5(1):e8647. doi: 10.1371/journal.pone.0008647.


Immortalized Muscle Cell Model to Test the Exon Skipping Efficacy for Duchenne Muscular Dystrophy.

Nguyen Q, Yokota T.

J Pers Med. 2017 Oct 16;7(4). pii: E13. doi: 10.3390/jpm7040013. Review.


Recent advances in innovative therapeutic approaches for Duchenne muscular dystrophy: from discovery to clinical trials.

Shimizu-Motohashi Y, Miyatake S, Komaki H, Takeda S, Aoki Y.

Am J Transl Res. 2016 Jun 15;8(6):2471-89. eCollection 2016. Review.


The golden retriever model of Duchenne muscular dystrophy.

Kornegay JN.

Skelet Muscle. 2017 May 19;7(1):9. doi: 10.1186/s13395-017-0124-z. Review.

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