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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.
Developing standardized corticosteroid treatment for Duchenne muscular dystrophy.
Guglieri M, Bushby K, McDermott MP, Hart KA, Tawil R, Martens WB, Herr BE, McColl E, Wilkinson J, Kirschner J, King WM, Eagle M, Brown MW, Willis T, Hirtz D, Shieh PB, Straub V, Childs AM, Ciafaloni E, Butterfield RJ, Horrocks I, Spinty S, Flanigan KM, Kuntz NL, Baranello G, Roper H, Morrison L, Mah JK, Manzur AY, McDonald CM, Schara U, von der Hagen M, Barohn RJ, Campbell C, Darras BT, Finkel RS, Vita G, Hughes I, Mongini T, Pegoraro E, Wicklund M, Wilichowski E, Bryan Burnette W, Howard JF, McMillan HJ, Thangarajh M, Griggs RC. Guglieri M, et al. Among authors: thangarajh m. Contemp Clin Trials. 2017 Jul;58:34-39. doi: 10.1016/j.cct.2017.04.008. Epub 2017 Apr 24. Contemp Clin Trials. 2017. PMID: 28450193 Free PMC article. Clinical Trial.
A checklist for clinical trials in rare disease: obstacles and anticipatory actions-lessons learned from the FOR-DMD trial.
Crow RA, Hart KA, McDermott MP, Tawil R, Martens WB, Herr BE, McColl E, Wilkinson J, Kirschner J, King WM, Eagle M, Brown MW, Hirtz D, Lochmuller H, Straub V, Ciafaloni E, Shieh PB, Spinty S, Childs AM, Manzur AY, Morandi L, Butterfield RJ, Horrocks I, Roper H, Flanigan KM, Kuntz NL, Mah JK, Morrison L, Darras BT, von der Hagen M, Schara U, Wilichowski E, Mongini T, McDonald CM, Vita G, Barohn RJ, Finkel RS, Wicklund M, McMillan HJ Jr, Hughes I, Pegoraro E, Bryan Burnette W, Howard JF, Thangarajh M, Campbell C, Griggs RC, Bushby K, Guglieri M. Crow RA, et al. Among authors: thangarajh m. Trials. 2018 May 10;19(1):291. doi: 10.1186/s13063-018-2645-0. Trials. 2018. PMID: 29793540 Free PMC article. Review.
249th ENMC International Workshop: The role of brain dystrophin in muscular dystrophy: Implications for clinical care and translational research, Hoofddorp, The Netherlands, November 29th-December 1st 2019.
Hendriksen JGM, Thangarajh M, Kan HE, Muntoni F; ENMC 249th workshop study group. Hendriksen JGM, et al. Among authors: thangarajh m. Neuromuscul Disord. 2020 Sep;30(9):782-794. doi: 10.1016/j.nmd.2020.08.357. Epub 2020 Aug 15. Neuromuscul Disord. 2020. PMID: 32912717 Free article. No abstract available.
Effect of Different Corticosteroid Dosing Regimens on Clinical Outcomes in Boys With Duchenne Muscular Dystrophy: A Randomized Clinical Trial.
Guglieri M, Bushby K, McDermott MP, Hart KA, Tawil R, Martens WB, Herr BE, McColl E, Speed C, Wilkinson J, Kirschner J, King WM, Eagle M, Brown MW, Willis T, Griggs RC; FOR-DMD Investigators of the Muscle Study Group; Straub V, van Ruiten H, Childs AM, Ciafaloni E, Shieh PB, Spinty S, Maggi L, Baranello G, Butterfield RJ, Horrocks IA, Roper H, Alhaswani Z, Flanigan KM, Kuntz NL, Manzur A, Darras BT, Kang PB, Morrison L, Krzesniak-Swinarska M, Mah JK, Mongini TE, Ricci F, von der Hagen M, Finkel RS, O'Reardon K, Wicklund M, Kumar A, McDonald CM, Han JJ, Joyce N, Henricson EK, Schara-Schmidt U, Gangfuss A, Wilichowski E, Barohn RJ, Statland JM, Campbell C, Vita G, Vita GL, Howard JF Jr, Hughes I, McMillan HJ, Pegoraro E, Bello L, Burnette WB, Thangarajh M, Chang T. Guglieri M, et al. Among authors: thangarajh m. JAMA. 2022 Apr 19;327(15):1456-1468. doi: 10.1001/jama.2022.4315. JAMA. 2022. PMID: 35381069 Free PMC article. Clinical Trial.
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.
50 results