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Page 1
Debamestrocel multimodal effects on biomarker pathways in amyotrophic lateral sclerosis are linked to clinical outcomes.
Lindborg SR, Goyal NA, Katz J, Burford M, Li J, Kaspi H, Abramov N, Boulanger B, Berry JD, Nicholson K, Mozaffar T, Miller R, Jenkins L, Baloh RH, Lewis R, Staff NP, Owegi MA, Dagher B, Blondheim-Shraga NR, Gothelf Y, Levy YS, Kern R, Aricha R, Windebank AJ, Bowser R, Brown RH Jr, Cudkowicz ME. Lindborg SR, et al. Among authors: goyal na. Muscle Nerve. 2024 Jun;69(6):719-729. doi: 10.1002/mus.28093. Epub 2024 Apr 9. Muscle Nerve. 2024. PMID: 38593477 Clinical Trial.
A randomized, placebo-controlled, double-blind phase IIb trial evaluating the safety and efficacy of tirasemtiv in patients with amyotrophic lateral sclerosis.
Shefner JM, Wolff AA, Meng L, Bian A, Lee J, Barragan D, Andrews JA; BENEFIT-ALS Study Group. Shefner JM, et al. Amyotroph Lateral Scler Frontotemporal Degener. 2016 Jul-Aug;17(5-6):426-435. doi: 10.3109/21678421.2016.1148169. Epub 2016 Mar 16. Amyotroph Lateral Scler Frontotemporal Degener. 2016. PMID: 26982815 Clinical Trial.
Long-term safety and efficacy of eculizumab in generalized myasthenia gravis.
Muppidi S, Utsugisawa K, Benatar M, Murai H, Barohn RJ, Illa I, Jacob S, Vissing J, Burns TM, Kissel JT, Nowak RJ, Andersen H, Casasnovas C, de Bleecker JL, Vu TH, Mantegazza R, O'Brien FL, Wang JJ, Fujita KP, Howard JF Jr; Regain Study Group. Muppidi S, et al. Muscle Nerve. 2019 Jul;60(1):14-24. doi: 10.1002/mus.26447. Epub 2019 Mar 8. Muscle Nerve. 2019. PMID: 30767274 Free PMC article.
A phase III trial of tirasemtiv as a potential treatment for amyotrophic lateral sclerosis.
Shefner JM, Cudkowicz ME, Hardiman O, Cockcroft BM, Lee JH, Malik FI, Meng L, Rudnicki SA, Wolff AA, Andrews JA; VITALITY-ALS Study Group. Shefner JM, et al. Amyotroph Lateral Scler Frontotemporal Degener. 2019;0(0):1-11. doi: 10.1080/21678421.2019.1612922. Amyotroph Lateral Scler Frontotemporal Degener. 2019. PMID: 31081694 Clinical Trial.
Design and Statistical Innovations in a Platform Trial for Amyotrophic Lateral Sclerosis.
Quintana M, Saville BR, Vestrucci M, Detry MA, Chibnik L, Shefner J, Berry JD, Chase M, Andrews J, Sherman AV, Yu H, Drake K, Cudkowicz M, Paganoni S, Macklin EA; HEALEY ALS Platform Trial Study Group. Quintana M, et al. Ann Neurol. 2023 Sep;94(3):547-560. doi: 10.1002/ana.26714. Epub 2023 Jun 22. Ann Neurol. 2023. PMID: 37245090
Safety and efficacy of arimoclomol in patients with early amyotrophic lateral sclerosis (ORARIALS-01): a randomised, double-blind, placebo-controlled, multicentre, phase 3 trial.
Benatar M, Hansen T, Rom D, Geist MA, Blaettler T, Camu W, Kuzma-Kozakiewicz M, van den Berg LH, Morales RJ, Chio A, Andersen PM, Pradat PF, Lange D, Van Damme P, Mora G, Grudniak M, Elliott M, Petri S, Olney N, Ladha S, Goyal NA, Meyer T, Hanna MG, Quinn C, Genge A, Zinman L, Jabari D, Shoesmith C, Ludolph AC, Neuwirth C, Nations S, Shefner JM, Turner MR, Wuu J, Bennett R, Dang H, Sundgreen C; ORARIALS-01 trial team. Benatar M, et al. Among authors: goyal na. Lancet Neurol. 2024 May 20:S1474-4422(24)00134-0. doi: 10.1016/S1474-4422(24)00134-0. Online ahead of print. Lancet Neurol. 2024. PMID: 38782015
Safety and efficacy of eculizumab in anti-acetylcholine receptor antibody-positive refractory generalised myasthenia gravis (REGAIN): a phase 3, randomised, double-blind, placebo-controlled, multicentre study.
Howard JF Jr, Utsugisawa K, Benatar M, Murai H, Barohn RJ, Illa I, Jacob S, Vissing J, Burns TM, Kissel JT, Muppidi S, Nowak RJ, O'Brien F, Wang JJ, Mantegazza R; REGAIN Study Group. Howard JF Jr, et al. Lancet Neurol. 2017 Dec;16(12):976-986. doi: 10.1016/S1474-4422(17)30369-1. Epub 2017 Oct 20. Lancet Neurol. 2017. PMID: 29066163 Clinical Trial.
60 results