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2,032 results

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Page 1
Safety and efficacy of losmapimod in facioscapulohumeral muscular dystrophy (ReDUX4): a randomised, double-blind, placebo-controlled phase 2b trial.
Tawil R, Wagner KR, Hamel JI, Leung DG, Statland JM, Wang LH, Genge A, Sacconi S, Lochmüller H, Reyes-Leiva D, Diaz-Manera J, Alonso-Perez J, Muelas N, Vilchez JJ, Pestronk A, Gibson S, Goyal NA, Hayward LJ, Johnson N, LoRusso S, Freimer M, Shieh PB, Subramony SH, van Engelen B, Kools J, Leinhard OD, Widholm P, Morabito C, Moxham CM, Cadavid D, Mellion ML, Odueyungbo A, Tracewell WG, Accorsi A, Ronco L, Gould RJ, Shoskes J, Rojas LA, Jiang JG. Tawil R, et al. Among authors: wang lh. Lancet Neurol. 2024 May;23(5):477-486. doi: 10.1016/S1474-4422(24)00073-5. Lancet Neurol. 2024. PMID: 38631764 Clinical Trial.
Facioscapulohumeral Dystrophy.
Wang LH, Tawil R. Wang LH, et al. Curr Neurol Neurosci Rep. 2016 Jul;16(7):66. doi: 10.1007/s11910-016-0667-0. Curr Neurol Neurosci Rep. 2016. PMID: 27215221 Review.
Defining SOD1 ALS natural history to guide therapeutic clinical trial design.
Bali T, Self W, Liu J, Siddique T, Wang LH, Bird TD, Ratti E, Atassi N, Boylan KB, Glass JD, Maragakis NJ, Caress JB, McCluskey LF, Appel SH, Wymer JP, Gibson S, Zinman L, Mozaffar T, Callaghan B, McVey AL, Jockel-Balsarotti J, Allred P, Fisher ER, Lopate G, Pestronk A, Cudkowicz ME, Miller TM. Bali T, et al. Among authors: wang lh. J Neurol Neurosurg Psychiatry. 2017 Feb;88(2):99-105. doi: 10.1136/jnnp-2016-313521. Epub 2016 Jun 3. J Neurol Neurosurg Psychiatry. 2017. PMID: 27261500 Free PMC article.
Clinical trial readiness to solve barriers to drug development in FSHD (ReSolve): protocol of a large, international, multi-center prospective study.
LoRusso S, Johnson NE, McDermott MP, Eichinger K, Butterfield RJ, Carraro E, Higgs K, Lewis L, Mul K, Sacconi S, Sansone VA, Shieh P, van Engelen B, Wagner K, Wang L, Statland JM, Tawil R; ReSolve Investigators and the FSHD CTRN18. LoRusso S, et al. BMC Neurol. 2019 Sep 10;19(1):224. doi: 10.1186/s12883-019-1452-x. BMC Neurol. 2019. PMID: 31506080 Free PMC article.
Longitudinal measures of RNA expression and disease activity in FSHD muscle biopsies.
Wong CJ, Wang LH, Friedman SD, Shaw D, Campbell AE, Budech CB, Lewis LM, Lemmers RJFL, Statland JM, van der Maarel SM, Tawil RN, Tapscott SJ. Wong CJ, et al. Among authors: wang lh. Hum Mol Genet. 2020 Apr 15;29(6):1030-1043. doi: 10.1093/hmg/ddaa031. Hum Mol Genet. 2020. PMID: 32083293 Free PMC article.
Effects of mexiletine on hyperexcitability in sporadic amyotrophic lateral sclerosis: Preliminary findings from a small phase II randomized controlled trial.
Weiss MD, Macklin EA, McIlduff CE, Vucic S, Wainger BJ, Kiernan MC, Goutman SA, Goyal NA, Rutkove SB, Ladha SS, Chen IA, Harms MB, Brannagan TH, Lacomis D, Zivkovic S, Ma M, Wang LH, Simmons Z, Rivner MH, Shefner JM, Cudkowicz ME, Atassi N; Mexiletine-2 ALS Study Group. Weiss MD, et al. Among authors: wang lh. Muscle Nerve. 2021 Mar;63(3):371-383. doi: 10.1002/mus.27146. Epub 2020 Dec 31. Muscle Nerve. 2021. PMID: 33340120 Free PMC article. Clinical Trial.
2,032 results