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A Randomized, Double-Blind, Placebo-Controlled, Global Phase 3 Study of Edasalonexent in Pediatric Patients with Duchenne Muscular Dystrophy: Results of the PolarisDMD Trial.
J Neuromuscul Dis. 2021;8(5):769-784. doi: 10.3233/JND-210689.
J Neuromuscul Dis. 2021.
PMID: 34120912
Free PMC article.
Clinical Trial.
Phase 1 Study of Edasalonexent (CAT-1004), an Oral NF-κB Inhibitor, in Pediatric Patients with Duchenne Muscular Dystrophy.
Finanger E, Vandenborne K, Finkel RS, Lee Sweeney H, Tennekoon G, Yum S, Mancini M, Bista P, Nichols A, Liu H, Fretzen A, Donovan JM.
Finanger E, et al. Among authors: lee sweeney h.
J Neuromuscul Dis. 2019;6(1):43-54. doi: 10.3233/JND-180341.
J Neuromuscul Dis. 2019.
PMID: 30452422
Free PMC article.
Clinical Trial.
Item in Clipboard
Ataluren treatment of patients with nonsense mutation dystrophinopathy.
Bushby K, Finkel R, Wong B, Barohn R, Campbell C, Comi GP, Connolly AM, Day JW, Flanigan KM, Goemans N, Jones KJ, Mercuri E, Quinlivan R, Renfroe JB, Russman B, Ryan MM, Tulinius M, Voit T, Moore SA, Lee Sweeney H, Abresch RT, Coleman KL, Eagle M, Florence J, Gappmaier E, Glanzman AM, Henricson E, Barth J, Elfring GL, Reha A, Spiegel RJ, O'donnell MW, Peltz SW, Mcdonald CM; PTC124-GD-007-DMD STUDY GROUP.
Bushby K, et al. Among authors: lee sweeney h.
Muscle Nerve. 2014 Oct;50(4):477-87. doi: 10.1002/mus.24332.
Muscle Nerve. 2014.
PMID: 25042182
Free PMC article.
Clinical Trial.
Item in Clipboard
Relationships of thigh muscle contractile and non-contractile tissue with function, strength, and age in boys with Duchenne muscular dystrophy.
Akima H, Lott D, Senesac C, Deol J, Germain S, Arpan I, Bendixen R, Lee Sweeney H, Walter G, Vandenborne K.
Akima H, et al. Among authors: lee sweeney h.
Neuromuscul Disord. 2012 Jan;22(1):16-25. doi: 10.1016/j.nmd.2011.06.750. Epub 2011 Jul 31.
Neuromuscul Disord. 2012.
PMID: 21807516
Free PMC article.
Item in Clipboard
Assessment of intramuscular lipid and metabolites of the lower leg using magnetic resonance spectroscopy in boys with Duchenne muscular dystrophy.
Lott DJ, Forbes SC, Mathur S, Germain SA, Senesac CR, Lee Sweeney H, Walter GA, Vandenborne K.
Lott DJ, et al. Among authors: lee sweeney h.
Neuromuscul Disord. 2014 Jul;24(7):574-82. doi: 10.1016/j.nmd.2014.03.013. Epub 2014 Apr 13.
Neuromuscul Disord. 2014.
PMID: 24798221
Free PMC article.
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Percutaneous Transendocardial Delivery of Self-complementary Adeno-associated Virus 6 Achieves Global Cardiac Gene Transfer in Canines.
Bish LT, Sleeper MM, Brainard B, Cole S, Russell N, Withnall E, Arndt J, Reynolds C, Davison E, Sanmiguel J, Wu D, Gao G, Wilson JM, Lee Sweeney H.
Bish LT, et al. Among authors: lee sweeney h.
Mol Ther. 2008 Dec;16(12):1953-1959. doi: 10.1038/mt.2008.202. Epub 2016 Dec 8.
Mol Ther. 2008.
PMID: 28189009
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