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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. 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. Muscle Nerve. 2025 Aug;72(2):294-305 PMID: 40420561 SCOPUS ID: 2-s2.0-105007077617 05/27/2025
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. Reldesemtiv in Amyotrophic Lateral Sclerosis: Results From the COURAGE-ALS Randomized Clinical Trial. JAMA Neurol. 2025 May 01;82(5):477-485 PMID: 40126464 PMCID: PMC11933997 SCOPUS ID: 2-s2.0-105002743538 03/24/2025
Writing Committee for the HEALEY ALS Platform Trial, Berry JD, Maragakis NJ, Macklin EA, Chibnik LB, Quintana M, Saville BR, Detry MA, Vestrucci M, Marion J, McGlothlin A, Stommel EW, Chase M, Pothier L, Harkey BA, Yu H, Sherman A, Shefner J, Hall M, Kittle G, Babu S, Andrews J, D\'Agostino D, Tustison E, Scirocco E, Giacomelli E, Alameda G, Locatelli E, Ho D, Quick A, Ajroud-Driss S, Katz J, Heitzman D, Appel SH, Shroff S, Felice KJ, Simmons Z, Miller T, Olney N, Weiss MD, Goutman SA, Fernandes JA Jr, Jawdat O, Owegi MA, Foster L, Vu T, Ilieva H, Newman DS, Arcila-Londono X, Jackson C, Ladha S, Heiman-Patterson T, Caress J, Swenson A, Peltier A, Lewis R, Fee D, Elliott M, Bedlack R, Kasarskis EJ, Elman L, Rosenfeld J, Walk D, McIlduff CE, Twydell P, Young E, Johnson K, Rezania K, Goyal NA, Cohen JA, Benatar M, Jones V, Glass J, Shah J, Beydoun SR, Wymer JP, Zilliox L, Nayar S, Pattee GL, Martinez-Thompson J, Rynders A, Evan J, Evan J, Hartford A, Sepassi M, Ho KS, Glanzman R, Greenberg B, Hotchkin MT, Paganoni S, Cudkowicz ME, HEALEY ALS Platform Trial Study Group. CNM-Au8 in Amyotrophic Lateral Sclerosis: The HEALEY ALS Platform Trial. JAMA. 2025 Feb 17;333(13):1138-49 PMID: 40067821 PMCID: PMC11833661 SCOPUS ID: 2-s2.0-105003032316 03/12/2025
Writing Committee for the HEALEY ALS Platform Trial, Andrews J, Paganoni S, Macklin EA, Chibnik LB, Quintana M, Saville BR, Detry MA, Vestrucci M, Marion J, McGlothlin A, Young E, Chase M, Pothier L, Harkey B, Yu H, Sherman A, Shefner J, Hall M, Kittle G, Connolly MR, Berry JD, D\'Agostino D, Tustison E, Giacomelli E, Scirocco E, Alameda G, Locatelli E, Ho D, Quick A, Heitzman D, Ajroud-Driss S, Appel SH, Shroff S, Katz J, Felice K, Maragakis NJ, Simmons Z, Goutman SA, Olney N, Miller T, Fernandes JA, Ilieva H, Jawdat O, Weiss MD, Foster L, Vu T, Ladha S, Owegi MA, Newman DS, Arcila-Londono X, Jackson CE, Swenson A, Heiman-Patterson T, Caress J, Fee D, Peltier A, Lewis R, Rosenfeld J, Walk D, Johnson K, Elliott M, Kasarskis EJ, Rutkove S, McIlduff CE, Bedlack R, Elman L, Goyal NA, Rezania K, Twydell P, Benatar M, Glass J, Cohen JA, Jones V, Zilliox L, Wymer JP, Beydoun SR, Shah J, Pattee GL, Martinez-Thompson J, Nayar S, Granit V, Donohue M, Grossman K, Campbell DJ, Qureshi IA, Cudkowicz ME, Babu S. Verdiperstat in Amyotrophic Lateral Sclerosis: Results From the Randomized HEALEY ALS Platform Trial. JAMA Neurol. 2025 Apr;82(4):333-343 PMID: 40067754 PMCID: PMC11833655 SCOPUS ID: 2-s2.0-105003693721 03/12/2025
Writing Committee for the HEALEY ALS Platform Trial, Shefner JM, Oskarsson B, Macklin EA, Chibnik LB, Quintana M, Saville BR, Detry MA, Vestrucci M, Marion J, McGlothlin A, Heiman-Patterson T, Chase M, Pothier L, Harkey BA, Yu H, Sherman AV, Hall M, Kittle G, Berry JD, Babu S, Andrews J, D\'Agostino D, Tustison E, Scirocco E, Giacomelli E, Alameda G, Locatelli E, Ho D, Quick A, Ajroud-Driss S, Katz J, Heitzman D, Appel SH, Shroff S, Felice K, Maragakis NJ, Simmons Z, Miller TM, Olney N, Weiss MD, Goutman SA, Fernandes JA, Jawdat O, Owegi MA, Foster LA, Vu T, Ilieva H, Newman DS, Arcila-Londono X, Jackson CE, Ladha S, Caress JB, Swenson A, Peltier A, Lewis RA, Fee D, Elliott M, Bedlack R, Kasarskis EJ, Elman L, Rosenfeld J, Walk D, McIlduff C, Twydell P, Young E, Johnson K, Rezania K, Goyal NA, Cohen JA, Benatar M, Jones V, Shah J, Beydoun SR, Wymer JP, Zilliox L, Nayar S, Pattee GL, Martinez-Thompson J, Leitner ML, Chen K, Goldberg YP, Cohen Y, Geva M, Hayden MR, Paganoni S, Cudkowicz ME, HEALEY ALS Platform Trial Study Group. Pridopidine in Amyotrophic Lateral Sclerosis: The HEALEY ALS Platform Trial. JAMA. 2025 Feb 17;333(13):1128-37 PMID: 40067755 PMCID: PMC11833658 SCOPUS ID: 2-s2.0-105003026814 03/12/2025
Paganoni S, Fournier CN, Macklin EA, Chibnik LB, Quintana M, Saville BR, Detry MA, Vestrucci M, Marion J, McGlothlin A, Ajroud-Driss S, Chase M, Pothier L, Harkey BA, Yu H, Sherman AV, Shefner JM, Hall M, Kittle G, Berry JD, Babu S, Andrews J, Dagostino D, Tustison E, Giacomelli E, Scirocco E, Alameda G, Locatelli E, Ho D, Quick A, Katz J, Heitzman D, Appel SH, Shroff S, Felice K, Maragakis NJ, Simmons Z, Miller TM, Olney N, Weiss MD, Goutman SA, Fernandes JA, Jawdat O, Owegi MA, Foster LA, Vu T, Ilieva H, Newman DS, Arcila-Londono X, Jackson CE, Ladha S, Heiman-Patterson T, Caress JB, Swenson A, Peltier A, Lewis R, Fee D, Elliott M, Bedlack R, Kasarskis EJ, Elman L, Rosenfeld J, Walk D, McIlduff C, Twydell P, Young E, Johnson K, Rezania K, Goyal NA, Cohen JA, Benatar M, Jones V, Glass J, Shah J, Beydoun SR, Wymer JP, Zilliox L, Nayar S, Pattee GL, Martinez-Thompson J, Harvey B, Patel S, Mahoney P, Duda PW, Cudkowicz ME, HEALEY ALS Platform Trial Study Group. Efficacy and Safety of Zilucoplan in Amyotrophic Lateral Sclerosis: A Randomized Clinical Trial. JAMA Netw Open. 2025 Feb 03;8(2):e2459058 PMID: 39960672 PMCID: PMC11833520 SCOPUS ID: 2-s2.0-85218953640 02/17/2025
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. Design and Statistical Innovations in a Platform Trial for Amyotrophic Lateral Sclerosis. Ann Neurol. 2023 Sep;94(3):547-560 PMID: 37245090 SCOPUS ID: 2-s2.0-85163197066 05/28/2023
Paganoni S, Berry JD, Quintana M, Macklin E, Saville BR, Detry MA, Chase M, Sherman AV, Yu H, Drake K, Andrews J, Shefner J, Chibnik LB, Vestrucci M, Cudkowicz ME, Macklin E, Quintana M, Saville BR, Detry MA, Chibnik LB, Bind MA, Chan J, Vestrucci M, Locatelli E, Alameda G, Paganoni S, Cudkowicz M, McCaffrey A, Berry J, Babu S, Nicholson K, Scalia J, Simmons Z, Grogan J, Su X, Mamarabadi M, Heitzman D, Nasri MA, Katz J, Jenkins L, Felice K, Whitaker C, Ajroud-Driss S, Quick A, Kolb S, Sarah Heintzman NP, Appel SH, Greene E, Thonhoff J, Shroff S, Liao B, Goutman SA, Feldman EL, Miller T, Amber Malcolm NP, Newman DS, Arcila-Londono X, Steijlen K, Owegi MA, Brown RH, Ghasemi M, Fernandes JAM, Piccione E, Thaisetthawatkul P, Weiss MD, Rad N, Ilieva H, Pasinelli P, Judith Guarnieri NP, Ladha S, Jacobsen B, Bowser R, Shefner J, Foster L, Jackson C, Rosenfeld J, Bhuvaneswaran K, Elman L, Quinn C, Julia Yasek NP, Fee D, Heiman-Patterson T, Deboo A, Swenson A, Nance C, Vu T, Suresh N, Farias J, Caress J, Cartwright M, Rezania K, Elliott M, Rutkove S, McIlduff C, Cohen JA, Stommel E, Glass J, Fournier C, Young E, Wymer JP. Adaptive Platform Trials to Transform Amyotrophic Lateral Sclerosis Therapy Development Annals of Neurology. 01 February 2022;91(2):165-175 SCOPUS ID: 2-s2.0-85122780992 02/01/2022
Cudkowicz M, Genge A, Maragakis N, Petri S, van den Berg L, Aho VV, Sarapohja T, Kuoppamäki M, Garratt C, Al-Chalabi A, Kiernan M, Mathers S, Henderson R, Needham M, Schultz D, Löscher W, Mitrovic N, Rath J, Damme PV, De Bleecker JL, Delstanche S, Johnston W, Zinman L, O\'Connell C, Matte G, Dionne A, Korngut L, Turnbull J, Laaksovirta H, Jokela M, Tapiola T, Soriani MH, Couratier P, Camu W, Corcia P, Ludolph A, Großkreutz J, Meyer T, Boentert M, Schrank B, Prudlo J, Untucht R, Hardiman O, Siciliano G, Chio\' A, Mazzini L, Inghilleri M, Caponnetto C, Mora G, Mora Pardina JS, Farrero Munoz E, Vázquez Costa JF, Aguera Morales E, Varona L, Andersen P, Ingre C, Johansson R, Radunovic A, Young C, Babu S, Shaibani A, Staff N, Vu T, Rivner M, Scelsa S, Sivakumar K, Waheed W, Heitzman D, Rana S, Pattee G, Ajroud-Driss S, Bayat E, Kasarskis E, Lange DJ, Elliott M, Harris B, Felice K, Pulley MT, Kwan J, Brown M, Ravits J, Burford M, Karam C, Miller T, Andrews J, Levine T, Locatelli E, Wymer J, Bedlack R, Fee D, Goyal N, Oskarsson B, McCluskey L, Caress J, Weiss M, Quick A, Bromberg M, Lacomis D, Goutman S, Rezania K. Safety and efficacy of oral levosimendan in people with amyotrophic lateral sclerosis (the REFALS study): a randomised, double-blind, placebo-controlled phase 3 trial Lancet Neurology. October 2021;20(10):821-831 SCOPUS ID: 2-s2.0-85114911882 10/01/2021