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 ME, 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 The Lancet Neurology. October 2021;20(10):821-831 SCOPUS ID: 2-s2.0-85114911882 10/01/2021
Shefner JM, Andrews JA, Genge A, Jackson C, Lechtzin N, Miller TM, Cockroft BM, Meng L, Wei J, Wolff AA, Malik FI, Bodkin C, Brooks BR, Caress J, Dionne A, Fee D, Goutman SA, Goyal NA, Hardiman O, Hayat G, Heiman-Patterson T, Heitzman D, Henderson RD, Johnston W, Karam C, Kiernan MC, Kolb SJ, Korngut L, Ladha S, Matte G, Mora JS, Needham M, Oskarsson B, Pattee GL, Pioro EP, Pulley M, Quan D, Rezania K, Schellenberg KL, Schultz D, Shoesmith C, Simmons Z, Statland J, Sultan S, Swenson A, Berg LHVD, Vu T, Vucic S, Weiss M, Whyte-Rayson A, Wymer J, Zinman L, Rudnicki SA. A Phase 2, Double-Blind, Randomized, Dose-Ranging Trial Of Reldesemtiv In Patients With ALS. Amyotroph Lateral Scler Frontotemporal Degener. 2021 May;22(3-4):287-299 PMID: 32969758 PMCID: PMC8117790 SCOPUS ID: 2-s2.0-85091435875 09/25/2020
Kavanaugh MS, Cho Y, Fee D, Barkhaus PE. Skill, confidence and support: conceptual elements of a child/youth caregiver training program in amyotrophic lateral sclerosis - the YCare protocol. Neurodegener Dis Manag. 2020 Aug;10(4):231-241 PMID: 32749175 SCOPUS ID: 2-s2.0-85095932496 08/05/2020
Čierny M, Hooshmand SI, Fee D, Tripathi S, Dsouza NR, La Pean Kirschner A, Zimmermann MT, Brennan R. Novel destabilizing Dynactin variant (DCTN1 p.Tyr78His) in patient with Perry syndrome. Parkinsonism Relat Disord. 2020 Aug;77:110-113 PMID: 32712562 SCOPUS ID: 2-s2.0-85088215494 07/28/2020
Kavanaugh MS, Cho CC, Howard M, Fee D, Barkhaus PE. US data on children and youth caregivers in amyotrophic lateral sclerosis. Neurology. 2020 Apr 07;94(14):e1452-e1459 PMID: 32188763 SCOPUS ID: 2-s2.0-85086092455 03/20/2020
Seminary ER, Santarriaga S, Wheeler L, Mejaki M, Abrudan J, Demos W, Zimmermann MT, Urrutia RA, Fee D, Barkhaus PE, Ebert AD. Motor Neuron Generation from iPSCs from Identical Twins Discordant for Amyotrophic Lateral Sclerosis. Cells. 2020 Feb 28;9(3) PMID: 32121108 PMCID: PMC7140469 SCOPUS ID: 2-s2.0-85092221701 03/04/2020
Raghavan M, Fee D, Barkhaus PE. Generation and propagation of the action potential. Handb Clin Neurol. 2019;160:3-22 PMID: 31277855 SCOPUS ID: 2-s2.0-85068161532 07/07/2019
Fee DB, Harmelink M, Monrad P, Pyzik E. Siblings With Mutations in TRAPPC11 Presenting With Limb-Girdle Muscular Dystrophy 2S. J Clin Neuromuscul Dis. 2017 Sep;19(1):27-30 PMID: 28827486 SCOPUS ID: 2-s2.0-85028630388 08/23/2017