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Items: 1 to 20 of 71

1.
2.

Improving clinical trial design for Duchenne muscular dystrophy.

Merlini L, Sabatelli P.

BMC Neurol. 2015 Aug 26;15:153. doi: 10.1186/s12883-015-0408-z.

3.

Advances in the Treatment of Duchenne Muscular Dystrophy: New and Emerging Pharmacotherapies.

Reinig AM, Mirzaei S, Berlau DJ.

Pharmacotherapy. 2017 Apr;37(4):492-499. doi: 10.1002/phar.1909. Epub 2017 Mar 10. Review.

PMID:
28152217
4.

Phase 2a study of ataluren-mediated dystrophin production in patients with nonsense mutation Duchenne muscular dystrophy.

Finkel RS, Flanigan KM, Wong B, Bönnemann C, Sampson J, Sweeney HL, Reha A, Northcutt VJ, Elfring G, Barth J, Peltz SW.

PLoS One. 2013 Dec 11;8(12):e81302. doi: 10.1371/journal.pone.0081302. eCollection 2013.

5.

Muscle dysfunction and structural defects of dystrophin-null sapje mutant zebrafish larvae are rescued by ataluren treatment.

Li M, Andersson-Lendahl M, Sejersen T, Arner A.

FASEB J. 2014 Apr;28(4):1593-9. doi: 10.1096/fj.13-240044. Epub 2013 Dec 26.

6.

The "rescue" of dystrophin synthesis in boys with Duchenne muscular dystrophy.

Nicholson LV.

Neuromuscul Disord. 1993 Sep-Nov;3(5-6):525-31.

PMID:
8186705
7.

Splice modification to restore functional dystrophin synthesis in Duchenne muscular dystrophy.

Wilton SD, Fletcher S.

Curr Pharm Des. 2010;16(8):988-1001. Review.

PMID:
20041827
8.

[Molecular genetics of Duchenne/Becker muscular dystrophy].

Saito K, Ikeya K, Kondo E, Yamauchi A, Komine S, Fukuyama Y.

Nihon Rinsho. 1993 Sep;51(9):2420-7. Review. Japanese.

PMID:
8411723
9.

Functional significance of dystrophin positive fibres in Duchenne muscular dystrophy.

Nicholson LV, Johnson MA, Bushby KM, Gardner-Medwin D.

Arch Dis Child. 1993 May;68(5):632-6.

10.

Pharmacological strategies for muscular dystrophy.

Khurana TS, Davies KE.

Nat Rev Drug Discov. 2003 May;2(5):379-90. Review.

PMID:
12750741
11.

[Mutation-specific treatments for Duchenne muscular dystrophy].

Matsuo M, Takeshima Y.

Brain Nerve. 2009 Aug;61(8):915-22. Review. Japanese.

PMID:
19697880
12.

Deficiency of brain synaptic dystrophin in human Duchenne muscular dystrophy.

Kim TW, Wu K, Black IB.

Ann Neurol. 1995 Sep;38(3):446-9.

PMID:
7668831
13.

Long-Term Efficacy, Safety, and Pharmacokinetics of Drisapersen in Duchenne Muscular Dystrophy: Results from an Open-Label Extension Study.

Goemans NM, Tulinius M, van den Hauwe M, Kroksmark AK, Buyse G, Wilson RJ, van Deutekom JC, de Kimpe SJ, Lourbakos A, Campion G.

PLoS One. 2016 Sep 2;11(9):e0161955. doi: 10.1371/journal.pone.0161955. eCollection 2016.

14.

Nanotherapy for Duchenne muscular dystrophy.

Nance ME, Hakim CH, Yang NN, Duan D.

Wiley Interdiscip Rev Nanomed Nanobiotechnol. 2017 Apr 11. doi: 10.1002/wnan.1472. [Epub ahead of print] Review.

PMID:
28398005
15.

Duchenne muscular dystrophy: deficiency of dystrophin at the muscle cell surface.

Bonilla E, Samitt CE, Miranda AF, Hays AP, Salviati G, DiMauro S, Kunkel LM, Hoffman EP, Rowland LP.

Cell. 1988 Aug 12;54(4):447-52.

PMID:
3042151
16.

Therapeutic restoration of dystrophin expression in Duchenne muscular dystrophy.

Wells DJ.

J Muscle Res Cell Motil. 2006;27(5-7):387-98. Epub 2006 Jul 28. Review.

PMID:
16874449
17.

The molecular and biochemical basis of Duchenne muscular dystrophy.

Anderson MS, Kunkel LM.

Trends Biochem Sci. 1992 Aug;17(8):289-92. Review.

PMID:
1412702
18.

Quantitative estimation of dystrophin protein: a sensitive and convenient "two-antibody sandwich" ELISA.

Ishikawa Y, Ishikawa Y, Minami R.

Tohoku J Exp Med. 1996 Sep;180(1):57-63.

19.
20.

Mosaic pattern of dystrophins in Duchenne muscular dystrophy.

Tachi N, Sasaki K, Yamada T, Imamura S, Mike T.

Pediatr Neurol. 1990 Jan-Feb;6(1):54-6.

PMID:
2178618

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