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Items: 1 to 20 of 26


Evidence for ACTN3 as a genetic modifier of Duchenne muscular dystrophy.

Hogarth MW, Houweling PJ, Thomas KC, Gordish-Dressman H, Bello L; Cooperative International Neuromuscular Research Group (CINRG)., Pegoraro E, Hoffman EP, Head SI, North KN.

Nat Commun. 2017 Jan 31;8:14143. doi: 10.1038/ncomms14143.


Nutraceuticals and Their Potential to Treat Duchenne Muscular Dystrophy: Separating the Credible from the Conjecture.

Woodman KG, Coles CA, Lamandé SR, White JD.

Nutrients. 2016 Nov 9;8(11). pii: E713. Review.


Spontaneous Breathing Pattern as Respiratory Functional Outcome in Children with Spinal Muscular Atrophy (SMA).

LoMauro A, Aliverti A, Mastella C, Arnoldi MT, Banfi P, Baranello G.

PLoS One. 2016 Nov 7;11(11):e0165818. doi: 10.1371/journal.pone.0165818.


Current and emerging treatment strategies for Duchenne muscular dystrophy.

Mah JK.

Neuropsychiatr Dis Treat. 2016 Jul 22;12:1795-807. doi: 10.2147/NDT.S93873. Review.


Hypoxia-induced cardiac injury in dystrophic mice.

Stelter Z, Strakova J, Yellamilli A, Fischer K, Sharpe K, Townsend D.

Am J Physiol Heart Circ Physiol. 2016 Apr 1;310(7):H938-48. doi: 10.1152/ajpheart.00917.2015.


Spell Checking Nature: Versatility of CRISPR/Cas9 for Developing Treatments for Inherited Disorders.

Wojtal D, Kemaladewi DU, Malam Z, Abdullah S, Wong TW, Hyatt E, Baghestani Z, Pereira S, Stavropoulos J, Mouly V, Mamchaoui K, Muntoni F, Voit T, Gonorazky HD, Dowling JJ, Wilson MD, Mendoza-Londono R, Ivakine EA, Cohn RD.

Am J Hum Genet. 2016 Jan 7;98(1):90-101. doi: 10.1016/j.ajhg.2015.11.012.


Histologic muscular history in steroid-treated and untreated patients with Duchenne dystrophy.

Peverelli L, Testolin S, Villa L, D'Amico A, Petrini S, Favero C, Magri F, Morandi L, Mora M, Mongini T, Bertini E, Sciacco M, Comi GP, Moggio M.

Neurology. 2015 Nov 24;85(21):1886-93. doi: 10.1212/WNL.0000000000002147.


Assessment and management of respiratory function in patients with Duchenne muscular dystrophy: current and emerging options.

LoMauro A, D'Angelo MG, Aliverti A.

Ther Clin Risk Manag. 2015 Sep 28;11:1475-88. doi: 10.2147/TCRM.S55889. Review.


What can Duchenne Connect teach us about treating Duchenne muscular dystrophy?

Wang RT, Nelson SF.

Curr Opin Neurol. 2015 Oct;28(5):535-41. doi: 10.1097/WCO.0000000000000245. Review.


Diastolic dysfunction precedes hypoxia-induced mortality in dystrophic mice.

Townsend D.

Physiol Rep. 2015 Aug;3(8). pii: e12513. doi: 10.14814/phy2.12513.


Duchenne muscular dystrophy: current cell therapies.

Sienkiewicz D, Kulak W, Okurowska-Zawada B, Paszko-Patej G, Kawnik K.

Ther Adv Neurol Disord. 2015 Jul;8(4):166-77. doi: 10.1177/1756285615586123. Review.


Online self-report data for duchenne muscular dystrophy confirms natural history and can be used to assess for therapeutic benefits.

Wang RT, Silverstein Fadlon CA, Ulm JW, Jankovic I, Eskin A, Lu A, Rangel Miller V, Cantor RM, Li N, Elashoff R, Martin AS, Peay HL, Halnon N, Nelson SF.

PLoS Curr. 2014 Oct 17;6. pii: doi: 10.1371/


Corticosteroid Treatments in Males With Duchenne Muscular Dystrophy: Treatment Duration and Time to Loss of Ambulation.

Kim S, Campbell KA, Fox DJ, Matthews DJ, Valdez R; MD STARnet..

J Child Neurol. 2015 Sep;30(10):1275-80. doi: 10.1177/0883073814558120.


Prevalence and distribution of late gadolinium enhancement in a large population of patients with Duchenne muscular dystrophy: effect of age and left ventricular systolic function.

Hor KN, Taylor MD, Al-Khalidi HR, Cripe LH, Raman SV, Jefferies JL, O'Donnell R, Benson DW, Mazur W.

J Cardiovasc Magn Reson. 2013 Dec 21;15:107. doi: 10.1186/1532-429X-15-107.


Novel compounds for the treatment of Duchenne muscular dystrophy: emerging therapeutic agents.

Wilton SD, Fletcher S.

Appl Clin Genet. 2011 Mar 10;4:29-44. doi: 10.2147/TACG.S8762.


The cooperative international neuromuscular research group Duchenne natural history study--a longitudinal investigation in the era of glucocorticoid therapy: design of protocol and the methods used.

McDonald CM, Henricson EK, Abresch RT, Han JJ, Escolar DM, Florence JM, Duong T, Arrieta A, Clemens PR, Hoffman EP, Cnaan A; Cinrg Investigators..

Muscle Nerve. 2013 Jul;48(1):32-54. doi: 10.1002/mus.23807.


Glucocorticoid-treated mice are an inappropriate positive control for long-term preclinical studies in the mdx mouse.

Sali A, Guerron AD, Gordish-Dressman H, Spurney CF, Iantorno M, Hoffman EP, Nagaraju K.

PLoS One. 2012;7(4):e34204. doi: 10.1371/journal.pone.0034204.

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