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181 results

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Page 1
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: shefner jm. Lancet Neurol. 2024 Jul;23(7):687-699. doi: 10.1016/S1474-4422(24)00134-0. Epub 2024 May 20. Lancet Neurol. 2024. PMID: 38782015 Clinical Trial.
Guidance for clinical management of pathogenic variant carriers at elevated genetic risk for ALS/FTD.
Benatar M, Heiman-Patterson TD, Cooper-Knock J, Brickman D, Casaletto KB, Goutman SA, Vinceti M, Dratch L, Arias JJ, Swidler J, Turner MR, Shefner J, Westeneng HJ, van den Berg LH, Al-Chalabi A; Attendees of the Workshop on Guidance for Clinical Care of People Living with a Pathogenic Variant At-Risk for ALS and FTD. Benatar M, et al. J Neurol Neurosurg Psychiatry. 2025 Jan 31;96(3):e334339. doi: 10.1136/jnnp-2024-334339. Online ahead of print. J Neurol Neurosurg Psychiatry. 2025. PMID: 39572211 Free PMC article. Review.
Randomized phase 2 trial of NP001-a novel immune regulator: Safety and early efficacy in ALS.
Miller RG, Block G, Katz JS, Barohn RJ, Gopalakrishnan V, Cudkowicz M, Zhang JR, McGrath MS, Ludington E, Appel SH, Azhir A; Phase 2 Trial NP001 Investigators. Miller RG, et al. Neurol Neuroimmunol Neuroinflamm. 2015 Apr 9;2(3):e100. doi: 10.1212/NXI.0000000000000100. eCollection 2015 Jun. Neurol Neuroimmunol Neuroinflamm. 2015. PMID: 25884010 Free PMC article.
Hospitalizations as an outcome measure in COURAGE-ALS.
Rudnicki SA, Al-Chalabi A, Andrews JA, Chio A, Corcia P, Couratier P, Cudkowicz ME, De Carvalho M, Genge A, Hardiman O, Heiman-Patterson T, Henderson RD, Ingre C, Johnston W, Ludolph A, Maragakis NJ, Miller TM, Mora JS, Petri S, Simmons Z, Van Den Berg LH, Zinman L, Herder KE, Kupfer S, Malik FI, Meng L, Simkins TJ, Wei J, Wolff AA, Shefner JM; Courage-Als study group. Rudnicki SA, et al. Among authors: shefner jm. Amyotroph Lateral Scler Frontotemporal Degener. 2025 Jun 12:1-10. doi: 10.1080/21678421.2025.2515907. Online ahead of print. Amyotroph Lateral Scler Frontotemporal Degener. 2025. PMID: 40503807
Operational Development and Launch of an Adaptive Platform Trial in Amyotrophic Lateral Sclerosis: Processes and Learnings From the First Four Regimens of the HEALEY ALS Platform Trial.
Harkey BA, Distefano S, Pagliaro JA, Heyd L, Chase M, Igne C, Yu H, Sherman AV, Dagostino D, Tustison E, Changkuon G, Hall M, Kittle G, Connolly MR, Giacomelli E, Scirocco E, Berry JD, Babu S, Shefner J, Macklin EA, Chibnik LB, De Mattos A, Drake K, Kamp C, McGarry A, Torti M, Small C, Bulat A, Cudkowicz ME, Paganoni S; HEALEY ALS Platform Trial Study Group. Harkey BA, et al. Muscle Nerve. 2025 May 26. doi: 10.1002/mus.28442. Online ahead of print. Muscle Nerve. 2025. PMID: 40420561
Reldesemtiv in Amyotrophic Lateral Sclerosis: Results From the COURAGE-ALS Randomized Clinical Trial.
Shefner JM, Cudkowicz ME, Genge A, Hardiman O, Al-Chalabi A, Andrews JA, Chio A, Corcia P, Couratier P, de Carvalho M, Heiman-Patterson T, Henderson RD, Ingre C, Johnston W, Ludolph A, Maragakis NJ, Miller TM, Mora JS, Petri S, Simmons Z, van den Berg LH, Zinman L, Kupfer S, Malik FI, Meng L, Simkins TJ, Wei J, Wolff AA, Rudnicki SA; COURAGE-ALS Study Group. Shefner JM, et al. JAMA Neurol. 2025 May 1;82(5):477-485. doi: 10.1001/jamaneurol.2025.0241. JAMA Neurol. 2025. PMID: 40126464 Free PMC article. Clinical Trial.
181 results