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Antisense oligonucleotide targeting DMPK in patients with myotonic dystrophy type 1: a multicentre, randomised, dose-escalation, placebo-controlled, phase 1/2a trial.
Thornton CA, Moxley RT 3rd, Eichinger K, Heatwole C, Mignon L, Arnold WD, Ashizawa T, Day JW, Dent G, Tanner MK, Duong T, Greene EP, Herbelin L, Johnson NE, King W, Kissel JT, Leung DG, Lott DJ, Norris DA, Pucillo EM, Schell W, Statland JM, Stinson N, Subramony SH, Xia S, Bishop KM, Bennett CF. Thornton CA, et al. Among authors: lott dj. Lancet Neurol. 2023 Mar;22(3):218-228. doi: 10.1016/S1474-4422(23)00001-7. Lancet Neurol. 2023. PMID: 36804094 Clinical Trial.
Examination of effects of corticosteroids on skeletal muscles of boys with DMD using MRI and MRS.
Arpan I, Willcocks RJ, Forbes SC, Finkel RS, Lott DJ, Rooney WD, Triplett WT, Senesac CR, Daniels MJ, Byrne BJ, Finanger EL, Russman BS, Wang DJ, Tennekoon GI, Walter GA, Sweeney HL, Vandenborne K. Arpan I, et al. Among authors: lott dj. Neurology. 2014 Sep 9;83(11):974-80. doi: 10.1212/WNL.0000000000000775. Epub 2014 Aug 6. Neurology. 2014. PMID: 25098537 Free PMC article. Clinical Trial.
Electrical impedance myography in individuals with collagen 6 and laminin α-2 congenital muscular dystrophy: a cross-sectional and 2-year analysis.
Nichols C, Jain MS, Meilleur KG, Wu T, Collins J, Waite MR, Dastgir J, Salman A, Donkervoort S, Duong T, Keller K, Leach ME, Lott DJ, McGuire MN, Nelson L, Rutkowski A, Vuillerot C, Bönnemann CG, Lehky TJ. Nichols C, et al. Among authors: lott dj. Muscle Nerve. 2018 Jan;57(1):54-60. doi: 10.1002/mus.25629. Epub 2017 Apr 25. Muscle Nerve. 2018. PMID: 28224647 Free PMC article.
Imaging respiratory muscle quality and function in Duchenne muscular dystrophy.
Barnard AM, Lott DJ, Batra A, Triplett WT, Forbes SC, Riehl SL, Willcocks RJ, Smith BK, Vandenborne K, Walter GA. Barnard AM, et al. Among authors: lott dj. J Neurol. 2019 Nov;266(11):2752-2763. doi: 10.1007/s00415-019-09481-z. Epub 2019 Jul 26. J Neurol. 2019. PMID: 31350642 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.
Modeling disease trajectory in Duchenne muscular dystrophy.
Rooney WD, Berlow YA, Triplett WT, Forbes SC, Willcocks RJ, Wang DJ, Arpan I, Arora H, Senesac C, Lott DJ, Tennekoon G, Finkel R, Russman BS, Finanger EL, Chakraborty S, O'Brien E, Moloney B, Barnard A, Sweeney HL, Daniels MJ, Walter GA, Vandenborne K. Rooney WD, et al. Among authors: lott dj. Neurology. 2020 Apr 14;94(15):e1622-e1633. doi: 10.1212/WNL.0000000000009244. Epub 2020 Mar 17. Neurology. 2020. PMID: 32184340 Free PMC article.
Clinical importance of changes in magnetic resonance biomarkers for Duchenne muscular dystrophy.
Willcocks RJ, Barnard AM, Daniels MJ, Forbes SC, Triplett WT, Brandsema JF, Finanger EL, Rooney WD, Kim S, Wang DJ, Lott DJ, Senesac CR, Walter GA, Sweeney HL, Vandenborne K. Willcocks RJ, et al. Among authors: lott dj. Ann Clin Transl Neurol. 2024 Jan;11(1):67-78. doi: 10.1002/acn3.51933. Epub 2023 Nov 6. Ann Clin Transl Neurol. 2024. PMID: 37932907 Free PMC article.
58 results