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Page 1
Patient reported outcomes in ALS: characteristics of the self-entry ALS Functional Rating Scale-revised and the Activities-specific Balance Confidence Scale.
Chew S, Burke KM, Collins E, Church R, Paganoni S, Nicholson K, Babu S, Scalia JB, De Marchi F, Ellrodt AL, Moura LMVR, Chan J, Berry JD. Chew S, et al. Among authors: scalia jb. Amyotroph Lateral Scler Frontotemporal Degener. 2021 Nov;22(7-8):467-477. doi: 10.1080/21678421.2021.1900259. Epub 2021 Mar 26. Amyotroph Lateral Scler Frontotemporal Degener. 2021. PMID: 33771057
Patient reported outcome measures (PROMs) in amyotrophic lateral sclerosis.
De Marchi F, Berry JD, Chan J, Caldwell S, Ellrodt A, Scalia J, Burke K, Fang T, Clark Sisodia R, Schwamm LH, Moura LMVR, Paganoni S. De Marchi F, et al. J Neurol. 2020 Jun;267(6):1754-1759. doi: 10.1007/s00415-020-09774-8. Epub 2020 Mar 3. J Neurol. 2020. PMID: 32125520
Adaptive Platform Trials to Transform Amyotrophic Lateral Sclerosis Therapy Development.
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; Healey ALS Platform Trial Study Group. Paganoni S, et al. Ann Neurol. 2022 Feb;91(2):165-175. doi: 10.1002/ana.26285. Epub 2022 Jan 10. Ann Neurol. 2022. PMID: 34935174 Review.
An expanded access protocol of RT001 in amyotrophic lateral sclerosis-Initial experience with a lipid peroxidation inhibitor.
Yerton M, Winter A, Kostov A, Lieberman C, Gelevski D, Weber H, Doyle M, Kane G, Parikh N, Burke KM, Rohrer M, Stirrat T, Bruno M, Hochman A, Luppino S, Scalia J, Skoniecki D, D'Agostino D, Sinani E, Yu H, Sherman AV, Babu S, Berry JD, Midei MG, Milner PG, Cudkowicz ME, Paganoni S. Yerton M, et al. Muscle Nerve. 2022 Oct;66(4):421-425. doi: 10.1002/mus.27672. Epub 2022 Jul 29. Muscle Nerve. 2022. PMID: 35765222 Free PMC article.
Trial of Antisense Oligonucleotide Tofersen for SOD1 ALS.
Miller TM, Cudkowicz ME, Genge A, Shaw PJ, Sobue G, Bucelli RC, Chiò A, Van Damme P, Ludolph AC, Glass JD, Andrews JA, Babu S, Benatar M, McDermott CJ, Cochrane T, Chary S, Chew S, Zhu H, Wu F, Nestorov I, Graham D, Sun P, McNeill M, Fanning L, Ferguson TA, Fradette S; VALOR and OLE Working Group. Miller TM, et al. N Engl J Med. 2022 Sep 22;387(12):1099-1110. doi: 10.1056/NEJMoa2204705. N Engl J Med. 2022. PMID: 36129998 Free article. Clinical Trial.
Safety and activity of anti-CD14 antibody IC14 (atibuclimab) in ALS: Experience with expanded access protocol.
Gelevski D, Addy G, Rohrer M, Cohen C, Roderick A, Winter A, Carey J, Scalia J, Yerton M, Weber H, Doyle M, Parikh N, Kane G, Ellrodt A, Burke K, D'Agostino D, Sinani E, Yu H, Sherman A, Agosti J, Redlich G, Charmley P, Crowe D, Appleby M, Ziegelaar B, Hanus K, Li Z, Babu S, Nicholson K, Luppino S, Berry J, Baecher-Allan C, Paganoni S, Cudkowicz M. Gelevski D, et al. Muscle Nerve. 2023 May;67(5):354-362. doi: 10.1002/mus.27775. Epub 2022 Dec 30. Muscle Nerve. 2023. PMID: 36533976
Expanded access protocol (EAP) program for access to investigational products for amyotrophic lateral sclerosis (ALS).
Yerton M, Winter A, Gelevski D, Addy G, Kostov A, Lieberman C, Weber H, Doyle M, Kane G, Cohen C, Parikh N, Burke KM, Rohrer M, Stirrat T, Bruno M, Hochman A, Luppino S, Scalia J, D'Agostino D, Sinani E, Yu H, Drake K, Hagar J, Sherman AV, Babu S, Berry JD, Cudkowicz ME, Paganoni S. Yerton M, et al. Muscle Nerve. 2023 Jun;67(6):456-463. doi: 10.1002/mus.27819. Epub 2023 Apr 5. Muscle Nerve. 2023. PMID: 36929648
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
Plasma metabolomic biomarker panel to distinguish patients with amyotrophic lateral sclerosis from disease mimics.
Lawton KA, Brown MV, Alexander D, Li Z, Wulff JE, Lawson R, Jaffa M, Milburn MV, Ryals JA, Bowser R, Cudkowicz ME, Berry JD; Northeast ALS Consortium. Lawton KA, et al. Amyotroph Lateral Scler Frontotemporal Degener. 2014 Sep;15(5-6):362-70. doi: 10.3109/21678421.2014.908311. Epub 2014 Jul 1. Amyotroph Lateral Scler Frontotemporal Degener. 2014. PMID: 24984169