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Page 1
Neurodevelopmental Needs in Young Boys with Duchenne Muscular Dystrophy (DMD): Observations from the Cooperative International Neuromuscular Research Group (CINRG) DMD Natural History Study (DNHS).
Thangarajh M, Spurney CF, Gordish-Dressman H, Clemens PR, Hoffman EP, McDonald CM, Henricson EK; CINRG Investigators. Thangarajh M, et al. PLoS Curr. 2018 Oct 17;10:ecurrents.md.4cdeb6970e54034db2bc3dfa54b4d987. doi: 10.1371/currents.md.4cdeb6970e54034db2bc3dfa54b4d987. PLoS Curr. 2018. PMID: 30443431 Free PMC article.
The NIH Toolbox for cognitive surveillance in Duchenne muscular dystrophy.
Thangarajh M, Kaat AJ, Bibat G, Mansour J, Summerton K, Gioia A, Berger C, Hardy KK, Wagner KR. Thangarajh M, et al. Ann Clin Transl Neurol. 2019 Sep;6(9):1696-1706. doi: 10.1002/acn3.50867. Epub 2019 Aug 31. Ann Clin Transl Neurol. 2019. PMID: 31472009 Free PMC article.
Relationships between DMD mutations and neurodevelopment in dystrophinopathy.
Thangarajh M, Hendriksen J, McDermott MP, Martens W, Hart KA, Griggs RC; Muscle Study Group and TREAT-NMD. Thangarajh M, et al. Neurology. 2019 Oct 22;93(17):e1597-e1604. doi: 10.1212/WNL.0000000000008363. Epub 2019 Oct 8. Neurology. 2019. PMID: 31594858 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.
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. Among authors: thangarajh m. 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.
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: thangarajh m. J Pers Med. 2020 Nov 19;10(4):236. doi: 10.3390/jpm10040236. J Pers Med. 2020. PMID: 33228131 Free PMC article.
Efficacy and Safety of Vamorolone in Duchenne Muscular Dystrophy: A 30-Month Nonrandomized Controlled Open-Label Extension Trial.
Mah JK, Clemens PR, Guglieri M, Smith EC, Finkel RS, Tulinius M, Nevo Y, Ryan MM, Webster R, Castro D, Kuntz NL, McDonald CM, Damsker JM, Schwartz BD, Mengle-Gaw LJ, Jackowski S, Stimpson G, Ridout DA, Ayyar-Gupta V, Baranello G, Manzur AY, Muntoni F, Gordish-Dressman H, Leinonen M, Ward LM, Hoffman EP, Dang UJ; NorthStar UK Network and CINRG DNHS Investigators. Mah JK, et al. JAMA Netw Open. 2022 Jan 4;5(1):e2144178. doi: 10.1001/jamanetworkopen.2021.44178. JAMA Netw Open. 2022. PMID: 35076703 Free PMC article. Clinical Trial.
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