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131 results

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Page 1
Multi-Omics Identifies Circulating miRNA and Protein Biomarkers for Facioscapulohumeral Dystrophy.
Heier CR, Zhang A, Nguyen NY, Tully CB, Panigrahi A, Gordish-Dressman H, Pandey SN, Guglieri M, Ryan MM, Clemens PR, Thangarajh M, Webster R, Smith EC, Connolly AM, McDonald CM, Karachunski P, Tulinius M, Harper A, Mah JK, Fiorillo AA, Chen YW, Cooperative International Neuromuscular Research Group Cinrg Investigators. Heier CR, et al. Among authors: clemens pr. J Pers Med. 2020 Nov 19;10(4):236. doi: 10.3390/jpm10040236. J Pers Med. 2020. PMID: 33228131 Free PMC article.
SPP1 genotype is a determinant of disease severity in Duchenne muscular dystrophy.
Pegoraro E, Hoffman EP, Piva L, Gavassini BF, Cagnin S, Ermani M, Bello L, Soraru G, Pacchioni B, Bonifati MD, Lanfranchi G, Angelini C, Kesari A, Lee I, Gordish-Dressman H, Devaney JM, McDonald CM; Cooperative International Neuromuscular Research Group. Pegoraro E, et al. Neurology. 2011 Jan 18;76(3):219-26. doi: 10.1212/WNL.0b013e318207afeb. Epub 2010 Dec 22. Neurology. 2011. PMID: 21178099 Free PMC article.
Randomized, blinded trial of weekend vs daily prednisone in Duchenne muscular dystrophy.
Escolar DM, Hache LP, Clemens PR, Cnaan A, McDonald CM, Viswanathan V, Kornberg AJ, Bertorini TE, Nevo Y, Lotze T, Pestronk A, Ryan MM, Monasterio E, Day JW, Zimmerman A, Arrieta A, Henricson E, Mayhew J, Florence J, Hu F, Connolly AM. Escolar DM, et al. Among authors: clemens pr. Neurology. 2011 Aug 2;77(5):444-52. doi: 10.1212/WNL.0b013e318227b164. Epub 2011 Jul 13. Neurology. 2011. PMID: 21753160 Free PMC article. Clinical Trial.
Vamorolone trial in Duchenne muscular dystrophy shows dose-related improvement of muscle function.
Hoffman EP, Schwartz BD, Mengle-Gaw LJ, Smith EC, Castro D, Mah JK, McDonald CM, Kuntz NL, Finkel RS, Guglieri M, Bushby K, Tulinius M, Nevo Y, Ryan MM, Webster R, Smith AL, Morgenroth LP, Arrieta A, Shimony M, Siener C, Jaros M, Shale P, McCall JM, Nagaraju K, van den Anker J, Conklin LS, Cnaan A, Gordish-Dressman H, Damsker JM, Clemens PR; Cooperative International Neuromuscular Research Group. Hoffman EP, et al. Among authors: clemens pr. Neurology. 2019 Sep 24;93(13):e1312-e1323. doi: 10.1212/WNL.0000000000008168. Epub 2019 Aug 26. Neurology. 2019. PMID: 31451516 Free PMC article. Clinical Trial.
TCTEX1D1 is a genetic modifier of disease progression in Duchenne muscular dystrophy.
Spitali P, Zaharieva I, Bohringer S, Hiller M, Chaouch A, Roos A, Scotton C, Claustres M, Bello L, McDonald CM, Hoffman EP; CINRG Investigators; Koeks Z, Eka Suchiman H, Cirak S, Scoto M, Reza M, 't Hoen PAC, Niks EH, Tuffery-Giraud S, Lochmüller H, Ferlini A, Muntoni F, Aartsma-Rus A. Spitali P, et al. Eur J Hum Genet. 2020 Jun;28(6):815-825. doi: 10.1038/s41431-019-0563-6. Epub 2020 Jan 2. Eur J Hum Genet. 2020. PMID: 31896777 Free PMC article.
Safety, Tolerability, and Efficacy of Viltolarsen in Boys With Duchenne Muscular Dystrophy Amenable to Exon 53 Skipping: A Phase 2 Randomized Clinical Trial.
Clemens PR, Rao VK, Connolly AM, Harper AD, Mah JK, Smith EC, McDonald CM, Zaidman CM, Morgenroth LP, Osaki H, Satou Y, Yamashita T, Hoffman EP; CINRG DNHS Investigators. Clemens PR, et al. JAMA Neurol. 2020 Aug 1;77(8):982-991. doi: 10.1001/jamaneurol.2020.1264. JAMA Neurol. 2020. PMID: 32453377 Free PMC article. Clinical Trial.
The CINRG Becker Natural History Study: Baseline characteristics.
Clemens PR, Niizawa G, Feng J, Florence J, DʼAlessandro AS, Morgenroth LP, Gorni K, Guglieri M, Connolly A, Wicklund M, Bertorini T, Mah JK, Thangarajh M, Smith E, Kuntz N, McDonald CM, Henricson EK, Upadhyayula S, Byrne B, Manousakis G, Harper A, Bravver E, Iannaccone S, Spurney C, Cnaan A, Gordish-Dressman H; CINRG BNHS Investigators. Clemens PR, et al. Muscle Nerve. 2020 Sep;62(3):369-376. doi: 10.1002/mus.27011. Epub 2020 Jul 24. Muscle Nerve. 2020. PMID: 32564389 Free PMC article.
Suitability of external controls for drug evaluation in Duchenne muscular dystrophy.
Goemans N, Signorovitch J, Sajeev G, Yao Z, Gordish-Dressman H, McDonald CM, Vandenborne K, Miller D, Ward SJ, Mercuri E; investigators from PRO-DMD-01 Study, CINRG DNHS, ImagingDMD, and the DMD Italian Group. Goemans N, et al. Neurology. 2020 Sep 8;95(10):e1381-e1391. doi: 10.1212/WNL.0000000000010170. Epub 2020 Jul 1. Neurology. 2020. PMID: 32611643 Free PMC article.
Efficacy and safety of vamorolone in Duchenne muscular dystrophy: An 18-month interim analysis of a non-randomized open-label extension study.
Smith EC, Conklin LS, Hoffman EP, Clemens PR, Mah JK, Finkel RS, Guglieri M, Tulinius M, Nevo Y, Ryan MM, Webster R, Castro D, Kuntz NL, Kerchner L, Morgenroth LP, Arrieta A, Shimony M, Jaros M, Shale P, Gordish-Dressman H, Hagerty L, Dang UJ, Damsker JM, Schwartz BD, Mengle-Gaw LJ, McDonald CM; CINRG VBP15 and DNHS Investigators. Smith EC, et al. Among authors: clemens pr. PLoS Med. 2020 Sep 21;17(9):e1003222. doi: 10.1371/journal.pmed.1003222. eCollection 2020 Sep. PLoS Med. 2020. PMID: 32956407 Free PMC article. Clinical Trial.
131 results