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Items: 1 to 50 of 162

1.

Clinical neurophysiology of anterior horn cell disorders.

Andrews JA, Shefner JM.

Handb Clin Neurol. 2019;161:317-326. doi: 10.1016/B978-0-444-64142-7.00057-6.

PMID:
31307610
2.

ALS drug development guidances and trial guidelines: Consensus and opportunities for alignment.

Andrews JA, Bruijn LI, Shefner JM.

Neurology. 2019 Jul 9;93(2):66-71. doi: 10.1212/WNL.0000000000007695. Epub 2019 Jun 6. Review.

3.

A phase III trial of tirasemtiv as a potential treatment for amyotrophic lateral sclerosis.

Shefner JM, Cudkowicz ME, Hardiman O, Cockroft BM, Lee JH, Malik FI, Meng L, Rudnicki SA, Wolff AA, Andrews JA; VITALITY-ALS STUDY GROUP.

Amyotroph Lateral Scler Frontotemporal Degener. 2019 May 13:1-11. doi: 10.1080/21678421.2019.1612922. [Epub ahead of print] Erratum in: Amyotroph Lateral Scler Frontotemporal Degener. 2019 Jul 14;:1.

PMID:
31081694
4.

Nusinersen in later-onset spinal muscular atrophy: Long-term results from the phase 1/2 studies.

Darras BT, Chiriboga CA, Iannaccone ST, Swoboda KJ, Montes J, Mignon L, Xia S, Bennett CF, Bishop KM, Shefner JM, Green AM, Sun P, Bhan I, Gheuens S, Schneider E, Farwell W, De Vivo DC; ISIS-396443-CS2/ISIS-396443-CS12 Study Groups.

Neurology. 2019 May 21;92(21):e2492-e2506. doi: 10.1212/WNL.0000000000007527. Epub 2019 Apr 24.

5.

ALS longitudinal studies with frequent data collection at home: study design and baseline data.

Rutkove SB, Qi K, Shelton K, Liss J, Berisha V, Shefner JM.

Amyotroph Lateral Scler Frontotemporal Degener. 2019 Feb;20(1-2):61-67. doi: 10.1080/21678421.2018.1541095. Epub 2018 Nov 28.

PMID:
30486680
6.

Relationships between slow vital capacity and measures of respiratory function on the ALSFRS-R.

Jackson C, De Carvalho M, Genge A, Heiman-Patterson T, Shefner JM, Wei J, Wolff AA.

Amyotroph Lateral Scler Frontotemporal Degener. 2018 Nov;19(7-8):506-512. doi: 10.1080/21678421.2018.1497658. Epub 2018 Oct 31.

PMID:
30379086
7.

Reducing sample size requirements for future ALS clinical trials with a dedicated electrical impedance myography system.

Shefner JM, Rutkove SB, Caress JB, Benatar M, David WS, Cartwright MS, Macklin EA, Bohorquez JL.

Amyotroph Lateral Scler Frontotemporal Degener. 2018 Nov;19(7-8):555-561. doi: 10.1080/21678421.2018.1510008. Epub 2018 Sep 28.

PMID:
30265154
8.

Effects of Strength Training in Amyotrophic Lateral Sclerosis: How Much Do We Know?

Shefner JM.

Muscle Nerve. 2019 Jan;59(1):6-7. doi: 10.1002/mus.26347. Epub 2018 Nov 28. No abstract available.

PMID:
30230558
9.

Edaravone in the treatment of amyotrophic lateral sclerosis: efficacy and access to therapy - a roundtable discussion.

Brooks BR, Jorgenson JA, Newhouse BJ, Shefner JM, Agnese W.

Am J Manag Care. 2018 Apr;24(9 Suppl):S175-S186.

10.

Assessment of bulbar function in amyotrophic lateral sclerosis: validation of a self-report scale (Center for Neurologic Study Bulbar Function Scale).

Smith RA, Macklin EA, Myers KJ, Pattee GL, Goslin KL, Meekins GD, Green JR, Shefner JM, Pioro EP.

Eur J Neurol. 2018 Jul;25(7):907-e66. doi: 10.1111/ene.13638. Epub 2018 Apr 16. Erratum in: Eur J Neurol. 2018 Oct;25(10):1303.

11.

Respiratory measures in amyotrophic lateral sclerosis.

Lechtzin N, Cudkowicz ME, de Carvalho M, Genge A, Hardiman O, Mitsumoto H, Mora JS, Shefner J, Van den Berg LH, Andrews JA.

Amyotroph Lateral Scler Frontotemporal Degener. 2018 Aug;19(5-6):321-330. doi: 10.1080/21678421.2018.1452945. Epub 2018 Mar 23. Erratum in: Amyotroph Lateral Scler Frontotemporal Degener. 2018 Aug;19(5-6):478.

PMID:
29566571
12.

VITALITY-ALS, a phase III trial of tirasemtiv, a selective fast skeletal muscle troponin activator, as a potential treatment for patients with amyotrophic lateral sclerosis: study design and baseline characteristics.

Andrews JA, Cudkowicz ME, Hardiman O, Meng L, Bian A, Lee J, Wolff AA, Malik FI, Shefner JM.

Amyotroph Lateral Scler Frontotemporal Degener. 2018 May;19(3-4):259-266. doi: 10.1080/21678421.2018.1426770. Epub 2018 Feb 6. Erratum in: Amyotroph Lateral Scler Frontotemporal Degener. 2018 May;19(3-4):321.

PMID:
29402141
13.

Association Between Decline in Slow Vital Capacity and Respiratory Insufficiency, Use of Assisted Ventilation, Tracheostomy, or Death in Patients With Amyotrophic Lateral Sclerosis.

Andrews JA, Meng L, Kulke SF, Rudnicki SA, Wolff AA, Bozik ME, Malik FI, Shefner JM.

JAMA Neurol. 2018 Jan 1;75(1):58-64. doi: 10.1001/jamaneurol.2017.3339.

14.

Profile of medical care costs in patients with amyotrophic lateral sclerosis in the Medicare programme and under commercial insurance.

Meng L, Bian A, Jordan S, Wolff A, Shefner JM, Andrews J.

Amyotroph Lateral Scler Frontotemporal Degener. 2018 Feb;19(1-2):134-142. doi: 10.1080/21678421.2017.1363242. Epub 2017 Sep 11.

PMID:
28891333
15.

Safety and efficacy of ozanezumab in patients with amyotrophic lateral sclerosis: a randomised, double-blind, placebo-controlled, phase 2 trial.

Meininger V, Genge A, van den Berg LH, Robberecht W, Ludolph A, Chio A, Kim SH, Leigh PN, Kiernan MC, Shefner JM, Desnuelle C, Morrison KE, Petri S, Boswell D, Temple J, Mohindra R, Davies M, Bullman J, Rees P, Lavrov A; NOG112264 Study Group.

Lancet Neurol. 2017 Mar;16(3):208-216. doi: 10.1016/S1474-4422(16)30399-4. Epub 2017 Jan 28.

16.

An Appraisal of Novel Biomarkers for Evaluating and Monitoring Neurologic Diseases: Editorial Introduction.

Shefner JM, Sabbagh MN.

Neurotherapeutics. 2017 Jan;14(1):1-3. doi: 10.1007/s13311-016-0502-y. No abstract available.

17.

Strength Testing in Motor Neuron Diseases.

Shefner JM.

Neurotherapeutics. 2017 Jan;14(1):154-160. doi: 10.1007/s13311-016-0472-0. Review.

18.

Quantitative strength testing in ALS clinical trials.

Shefner JM, Liu D, Leitner ML, Schoenfeld D, Johns DR, Ferguson T, Cudkowicz M.

Neurology. 2016 Aug 9;87(6):617-24. doi: 10.1212/WNL.0000000000002941. Epub 2016 Jul 6.

19.

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.

Amyotroph Lateral Scler Frontotemporal Degener. 2016 Jul-Aug;17(5-6):426-435. doi: 10.3109/21678421.2016.1148169. Epub 2016 Mar 16.

PMID:
26982815
20.

A randomized trial of mexiletine in ALS: Safety and effects on muscle cramps and progression.

Weiss MD, Macklin EA, Simmons Z, Knox AS, Greenblatt DJ, Atassi N, Graves M, Parziale N, Salameh JS, Quinn C, Brown RH Jr, Distad JB, Trivedi J, Shefner JM, Barohn RJ, Pestronk A, Swenson A, Cudkowicz ME; Mexiletine ALS Study Group.

Neurology. 2016 Apr 19;86(16):1474-81. doi: 10.1212/WNL.0000000000002507. Epub 2016 Feb 24.

21.

Preclinical Rodent Toxicity Studies for Long Term Use of Ceftriaxone.

Ratti E, Berry JD, Greenblatt DJ, Loci L, Ellrodt AS, Shefner JM, Cudkowicz ME.

Toxicol Rep. 2015;2:1396-1403.

22.

Reply.

Rutkove SB, Shefner JM, Bowser R, Benatar M.

Ann Neurol. 2016 Feb;79(2):334. doi: 10.1002/ana.24586. Epub 2016 Jan 19. No abstract available.

PMID:
26705262
23.

To travel or not to travel: The modern day struggle of the academic researcher.

Rutkove SB, Shefner JM, Bowser R, Benatar M.

Ann Neurol. 2015 Nov;78(5):667-9. doi: 10.1002/ana.24516. Epub 2015 Sep 18. No abstract available.

PMID:
26343928
24.

A revision of the El Escorial criteria - 2015.

Ludolph A, Drory V, Hardiman O, Nakano I, Ravits J, Robberecht W, Shefner J; WFN Research Group On ALS/MND.

Amyotroph Lateral Scler Frontotemporal Degener. 2015;16(5-6):291-2. doi: 10.3109/21678421.2015.1049183. Epub 2015 Jun 29. No abstract available.

PMID:
26121170
25.

The El Escorial criteria: strengths and weaknesses.

Agosta F, Al-Chalabi A, Filippi M, Hardiman O, Kaji R, Meininger V, Nakano I, Shaw P, Shefner J, van den Berg LH, Ludolph A; WFN Research Group on ALS/MND.

Amyotroph Lateral Scler Frontotemporal Degener. 2015 Mar;16(1-2):1-7. doi: 10.3109/21678421.2014.964258. Epub 2014 Dec 8.

PMID:
25482030
26.

Safety and efficacy of ceftriaxone for amyotrophic lateral sclerosis: a multi-stage, randomised, double-blind, placebo-controlled trial.

Cudkowicz ME, Titus S, Kearney M, Yu H, Sherman A, Schoenfeld D, Hayden D, Shui A, Brooks B, Conwit R, Felsenstein D, Greenblatt DJ, Keroack M, Kissel JT, Miller R, Rosenfeld J, Rothstein JD, Simpson E, Tolkoff-Rubin N, Zinman L, Shefner JM; Ceftriaxone Study Investigators.

Lancet Neurol. 2014 Nov;13(11):1083-1091. doi: 10.1016/S1474-4422(14)70222-4. Epub 2014 Oct 5.

27.

Quantifying disease progression in amyotrophic lateral sclerosis.

Simon NG, Turner MR, Vucic S, Al-Chalabi A, Shefner J, Lomen-Hoerth C, Kiernan MC.

Ann Neurol. 2014 Nov;76(5):643-57. doi: 10.1002/ana.24273. Epub 2014 Sep 30. Review.

28.

Assessment of disease progression and functional benefit in neurodegenerative disease: can we tell the difference?

Shefner JM, Mihaila D.

Amyotroph Lateral Scler Frontotemporal Degener. 2014 Sep;15(5-6):337-43. doi: 10.3109/21678421.2014.918150. Epub 2014 Jun 11. Review.

PMID:
24918689
29.

Safety, pharmacokinetic, and functional effects of the nogo-a monoclonal antibody in amyotrophic lateral sclerosis: a randomized, first-in-human clinical trial.

Meininger V, Pradat PF, Corse A, Al-Sarraj S, Rix Brooks B, Caress JB, Cudkowicz M, Kolb SJ, Lange D, Leigh PN, Meyer T, Milleri S, Morrison KE, Orrell RW, Peters G, Rothstein JD, Shefner J, Lavrov A, Williams N, Overend P, Price J, Bates S, Bullman J, Krull D, Berges A, Abila B, Meno-Tetang G, Wurthner J.

PLoS One. 2014 May 19;9(5):e97803. doi: 10.1371/journal.pone.0097803. eCollection 2014.

30.

Systemic pharmacokinetics and cerebrospinal fluid uptake of intravenous ceftriaxone in patients with amyotrophic lateral sclerosis.

Zhao Y, Cudkowicz ME, Shefner JM, Krivickas L, David WS, Vriesendorp F, Pestronk A, Caress JB, Katz J, Simpson E, Rosenfeld J, Pascuzzi R, Glass J, Rezania K, Harmatz JS, Schoenfeld D, Greenblatt DJ.

J Clin Pharmacol. 2014 Oct;54(10):1180-7. doi: 10.1002/jcph.317. Epub 2014 May 16. Erratum in: J Clin Pharmacol. 2014 Nov;54(11):1320.

31.

Predicting success: optimizing phase II ALS trials for the transition to phase III.

Berry JD, Cudkowicz ME, Shefner JM.

Amyotroph Lateral Scler Frontotemporal Degener. 2014 Mar;15(1-2):1-8. doi: 10.3109/21678421.2013.838969. Review.

PMID:
24588460
32.

ALS Multicenter Cohort Study of Oxidative Stress (ALS COSMOS): study methodology, recruitment, and baseline demographic and disease characteristics.

Mitsumoto H, Factor-Litvak P, Andrews H, Goetz RR, Andrews L, Rabkin JG, McElhiney M, Nieves J, Santella RM, Murphy J, Hupf J, Singleton J, Merle D, Kilty M, Heitzman D, Bedlack RS, Miller RG, Katz JS, Forshew D, Barohn RJ, Sorenson EJ, Oskarsson B, Fernandes Filho JA, Kasarskis EJ, Lomen-Hoerth C, Mozaffar T, Rollins YD, Nations SP, Swenson AJ, Shefner JM, Andrews JA, Koczon-Jaremko BA; ALS COSMOS Study Group.

Amyotroph Lateral Scler Frontotemporal Degener. 2014 Jun;15(3-4):192-203. doi: 10.3109/21678421.2013.864312. Epub 2014 Feb 24.

33.

Designing clinical trials for dystonia.

Galpern WR, Coffey CS, Albanese A, Cheung K, Comella CL, Ecklund DJ, Fahn S, Jankovic J, Kieburtz K, Lang AE, McDermott MP, Shefner JM, Teller JK, Thompson JL, Yeatts SD, Jinnah HA.

Neurotherapeutics. 2014 Jan;11(1):117-27. doi: 10.1007/s13311-013-0221-6. Review.

34.

Electrical impedance myography correlates with standard measures of ALS severity.

Rutkove SB, Caress JB, Cartwright MS, Burns TM, Warder J, David WS, Goyal N, Maragakis NJ, Benatar M, Sharma KR, Narayanaswami P, Raynor EM, Watson ML, Shefner JM.

Muscle Nerve. 2014 Mar;49(3):441-3. doi: 10.1002/mus.24128. Epub 2013 Dec 19.

PMID:
24273034
35.

Dexpramipexole versus placebo for patients with amyotrophic lateral sclerosis (EMPOWER): a randomised, double-blind, phase 3 trial.

Cudkowicz ME, van den Berg LH, Shefner JM, Mitsumoto H, Mora JS, Ludolph A, Hardiman O, Bozik ME, Ingersoll EW, Archibald D, Meyers AL, Dong Y, Farwell WR, Kerr DA; EMPOWER investigators.

Lancet Neurol. 2013 Nov;12(11):1059-67. doi: 10.1016/S1474-4422(13)70221-7. Epub 2013 Sep 23. Erratum in: Lancet Neurol. 2013 Nov;12(11):1042. Carbonell, J G [corrected to Gamez, J].

PMID:
24067398
36.

A study to evaluate safety and tolerability of repeated doses of tirasemtiv in patients with amyotrophic lateral sclerosis.

Shefner JM, Watson ML, Meng L, Wolff AA; Neals/Cytokinetics STUDY Team.

Amyotroph Lateral Scler Frontotemporal Degener. 2013 Dec;14(7-8):574-81. doi: 10.3109/21678421.2013.822517. Epub 2013 Aug 19.

PMID:
23952636
37.

The relationship between tirasemtiv serum concentration and functional outcomes in patients with ALS.

Shefner JM, Wolff AA, Meng L.

Amyotroph Lateral Scler Frontotemporal Degener. 2013 Dec;14(7-8):582-5. doi: 10.3109/21678421.2013.817587. Epub 2013 Aug 19.

PMID:
23952600
38.

Infrastructure resources for clinical research in amyotrophic lateral sclerosis.

Sherman AV, Gubitz AK, Al-Chalabi A, Bedlack R, Berry J, Conwit R, Harris BT, Horton DK, Kaufmann P, Leitner ML, Miller R, Shefner J, Vonsattel JP, Mitsumoto H.

Amyotroph Lateral Scler Frontotemporal Degener. 2013 May;14 Suppl 1:53-61. doi: 10.3109/21678421.2013.779058. Review.

PMID:
23678880
39.

Mechanisms, models and biomarkers in amyotrophic lateral sclerosis.

Turner MR, Bowser R, Bruijn L, Dupuis L, Ludolph A, McGrath M, Manfredi G, Maragakis N, Miller RG, Pullman SL, Rutkove SB, Shaw PJ, Shefner J, Fischbeck KH.

Amyotroph Lateral Scler Frontotemporal Degener. 2013 May;14 Suppl 1:19-32. doi: 10.3109/21678421.2013.778554. Review.

40.

ALS Untangled No. 20: the Deanna protocol.

ALSUntangled Group, Fournier C, Bedlack B, Hardiman O, Heiman-Patterson T, Gutmann L, Bromberg M, Ostrow L, Carter G, Kabashi E, Bertorini T, Mozaffar T, Andersen P, Dietz J, Gamez J, Dimachkie M, Wang Y, Wicks P, Heywood J, Novella S, Rowland LP, Pioro E, Kinsley L, Mitchell K, Glass J, Sathornsumetee S, Kwiecinski H, Baker J, Atassi N, Forshew D, Ravits J, Conwit R, Jackson C, Sherman A, Dalton K, Tindall K, Gonzalez G, Robertson J, Phillips L, Benatar M, Sorenson E, Shoesmith C, Nash S, Maragakis N, Moore D, Caress J, Boylan K, Armon C, Grosso M, Gerecke B, Wymer J, Oskarsson B, Bowser R, Drory V, Shefner J, Lechtzin N, Leitner M, Miller R, Mitsumoto H, Levine T, Russell J, Sharma K, Saperstein D, McClusky L, MacGowan D, Licht J, Verma A, Strong M, Lomen-Hoerth C, Tandan R, Rivner M, Kolb S, Polak M, Rudnicki S, Kittrell P, Quereshi M, Sachs G, Pattee G, Weiss M, Kissel J, Goldstein J, Rothstein J, Pastula D, Gleb L, Ogino M, Rosenfeld J, Carmi E, Oster C, Barkhaus P, Valor E.

Amyotroph Lateral Scler Frontotemporal Degener. 2013 May;14(4):319-23. doi: 10.3109/21678421.2013.788405. Epub 2013 May 2. Review. No abstract available.

PMID:
23638638
41.

Design and initial results of a multi-phase randomized trial of ceftriaxone in amyotrophic lateral sclerosis.

Berry JD, Shefner JM, Conwit R, Schoenfeld D, Keroack M, Felsenstein D, Krivickas L, David WS, Vriesendorp F, Pestronk A, Caress JB, Katz J, Simpson E, Rosenfeld J, Pascuzzi R, Glass J, Rezania K, Rothstein JD, Greenblatt DJ, Cudkowicz ME; Northeast ALS Consortium.

PLoS One. 2013 Apr 17;8(4):e61177. doi: 10.1371/journal.pone.0061177. Print 2013.

42.

Multimodal actions of neural stem cells in a mouse model of ALS: a meta-analysis.

Teng YD, Benn SC, Kalkanis SN, Shefner JM, Onario RC, Cheng B, Lachyankar MB, Marconi M, Li J, Yu D, Han I, Maragakis NJ, Lládo J, Erkmen K, Redmond DE Jr, Sidman RL, Przedborski S, Rothstein JD, Brown RH Jr, Snyder EY.

Sci Transl Med. 2012 Dec 19;4(165):165ra164. doi: 10.1126/scitranslmed.3004579.

PMID:
23253611
43.

Electrodiagnosis of motor neuron disease.

Duleep A, Shefner J.

Phys Med Rehabil Clin N Am. 2013 Feb;24(1):139-51. doi: 10.1016/j.pmr.2012.08.022. Epub 2012 Oct 25. Review.

PMID:
23177036
44.

Electrical impedance myography as a biomarker to assess ALS progression.

Rutkove SB, Caress JB, Cartwright MS, Burns TM, Warder J, David WS, Goyal N, Maragakis NJ, Clawson L, Benatar M, Usher S, Sharma KR, Gautam S, Narayanaswami P, Raynor EM, Watson ML, Shefner JM.

Amyotroph Lateral Scler. 2012 Sep;13(5):439-45. doi: 10.3109/17482968.2012.688837. Epub 2012 Jun 7.

45.

Safety, tolerability and pharmacodynamics of a skeletal muscle activator in amyotrophic lateral sclerosis.

Shefner J, Cedarbaum JM, Cudkowicz ME, Maragakis N, Lee J, Jones D, Watson ML, Mahoney K, Chen M, Saikali K, Mao J, Russell AJ, Hansen RL, Malik F, Wolff AA; Neals/Cytokinetics Study Team.

Amyotroph Lateral Scler. 2012 Sep;13(5):430-8. doi: 10.3109/17482968.2012.684214. Epub 2012 May 16.

PMID:
22591195
46.

The effects of dexpramipexole (KNS-760704) in individuals with amyotrophic lateral sclerosis.

Cudkowicz M, Bozik ME, Ingersoll EW, Miller R, Mitsumoto H, Shefner J, Moore DH, Schoenfeld D, Mather JL, Archibald D, Sullivan M, Amburgey C, Moritz J, Gribkoff VK.

Nat Med. 2011 Nov 20;17(12):1652-6. doi: 10.1038/nm.2579.

PMID:
22101764
47.

The Awaji criteria for diagnosis of ALS.

de Carvalho M, Dengler R, Eisen A, England JD, Kaji R, Kimura J, Mills K, Mitsumoto H, Nodera H, Shefner J, Swash M.

Muscle Nerve. 2011 Sep;44(3):456-7; author reply 457. doi: 10.1002/mus.22175. No abstract available.

PMID:
21996809
48.

Biomarkers in amyotrophic lateral sclerosis: opportunities and limitations.

Bowser R, Turner MR, Shefner J.

Nat Rev Neurol. 2011 Oct 11;7(11):631-8. doi: 10.1038/nrneurol.2011.151. Review.

PMID:
21989244
49.

Multipoint incremental motor unit number estimation as an outcome measure in ALS.

Shefner JM, Watson ML, Simionescu L, Caress JB, Burns TM, Maragakis NJ, Benatar M, David WS, Sharma KR, Rutkove SB.

Neurology. 2011 Jul 19;77(3):235-41. doi: 10.1212/WNL.0b013e318225aabf. Epub 2011 Jun 15.

50.

Characterizing spinal muscular atrophy with electrical impedance myography.

Rutkove SB, Shefner JM, Gregas M, Butler H, Caracciolo J, Lin C, Fogerson PM, Mongiovi P, Darras BT.

Muscle Nerve. 2010 Dec;42(6):915-21. doi: 10.1002/mus.21784.

PMID:
21104866

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