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Items: 1 to 20 of 239

1.

Progress in Drug Discovery in Academia and Persistent Challenges of "the Valley of Death".

Barohn RJ, Weir SJ, Simari RD.

Mayo Clin Proc. 2019 Mar;94(3):391-393. doi: 10.1016/j.mayocp.2019.01.014. No abstract available.

PMID:
30832788
2.

Safety, tolerability, pharmacokinetics, pharmacodynamics, and exploratory efficacy of the novel enzyme replacement therapy avalglucosidase alfa (neoGAA) in treatment-naïve and alglucosidase alfa-treated patients with late-onset Pompe disease: A phase 1, open-label, multicenter, multinational, ascending dose study.

Pena LDM, Barohn RJ, Byrne BJ, Desnuelle C, Goker-Alpan O, Ladha S, Laforêt P, Mengel KE, Pestronk A, Pouget J, Schoser B, Straub V, Trivedi J, Van Damme P, Vissing J, Young P, Kacena K, Shafi R, Thurberg BL, Culm-Merdek K, van der Ploeg AT; NEO1 Investigator Group.

Neuromuscul Disord. 2019 Mar;29(3):167-186. doi: 10.1016/j.nmd.2018.12.004. Epub 2018 Dec 17.

PMID:
30770310
3.

Long-term safety and efficacy of eculizumab in generalized myasthenia gravis.

Muppidi S, Utsugisawa K, Benatar M, Murai H, Barohn RJ, Illa I, Jacob S, Vissing J, Burns TM, Kissel JT, Nowak RJ, Andersen H, Casasnovas C, de Bleecker JL, Vu TH, Mantegazza R, O'Brien FL, Wang JJ, Fujita KP, Howard JF Jr; Regain Study Group.

Muscle Nerve. 2019 Feb 14. doi: 10.1002/mus.26447. [Epub ahead of print]

PMID:
30767274
4.

Long-term effect of thymectomy plus prednisone versus prednisone alone in patients with non-thymomatous myasthenia gravis: 2-year extension of the MGTX randomised trial.

Wolfe GI, Kaminski HJ, Aban IB, Minisman G, Kuo HC, Marx A, Ströbel P, Mazia C, Oger J, Cea JG, Heckmann JM, Evoli A, Nix W, Ciafaloni E, Antonini G, Witoonpanich R, King JO, Beydoun SR, Chalk CH, Barboi AC, Amato AA, Shaibani AI, Katirji B, Lecky BRF, Buckley C, Vincent A, Dias-Tosta E, Yoshikawa H, Waddington-Cruz M, Pulley MT, Rivner MH, Kostera-Pruszczyk A, Pascuzzi RM, Jackson CE, Verschuuren JJGM, Massey JM, Kissel JT, Werneck LC, Benatar M, Barohn RJ, Tandan R, Mozaffar T, Silvestri NJ, Conwit R, Sonett JR, Jaretzki A 3rd, Newsom-Davis J, Cutter GR; MGTX Study Group.

Lancet Neurol. 2019 Mar;18(3):259-268. doi: 10.1016/S1474-4422(18)30392-2. Epub 2019 Jan 25.

PMID:
30692052
5.

Thymectomy may not be associated with clinical improvement in MuSK myasthenia gravis.

Clifford KM, Hobson-Webb LD, Benatar M, Burns TM, Barnett C, Silvestri NJ, Howard JF Jr, Visser A, Crum BA, Nowak R, Beekman R, Kumar A, Ruzhansky K, Chen IA, Pulley MT, Laboy SM, Fellman MA, Howard DB, Kolb NA, Greene SM, Pasnoor M, Dimachkie MM, Barohn RJ, Hehir MK.

Muscle Nerve. 2019 Apr;59(4):404-410. doi: 10.1002/mus.26404. Epub 2019 Jan 14.

PMID:
30575980
6.

Using automated electronic medical record data extraction to model ALS survival and progression.

Karanevich AG, Weisbrod LJ, Jawdat O, Barohn RJ, Gajewski BJ, He J, Statland JM.

BMC Neurol. 2018 Dec 14;18(1):205. doi: 10.1186/s12883-018-1208-z.

7.

Satisfactory Response With Achieving Maintenance Low-Dose Prednisone in Generalized Myasthenia Gravis.

Abuzinadah AR, Jabari D, Jawdat O, Pasnoor M, Glenn M, Herbelin L, McVey AL, Barohn RJ, Dimachkie MM.

J Clin Neuromuscul Dis. 2018 Dec;20(2):49-59. doi: 10.1097/CND.0000000000000219.

PMID:
30439750
8.

Rasagiline for amyotrophic lateral sclerosis: A randomized, controlled trial.

Statland JM, Moore D, Wang Y, Walsh M, Mozaffar T, Elman L, Nations SP, Mitsumoto H, Fernandes JA, Saperstein D, Hayat G, Herbelin L, Karam C, Katz J, Wilkins HM, Agbas A, Swerdlow RH, Santella RM, Dimachkie MM, Barohn RJ; Rasagiline Investigators of the Muscle Study Group and Western ALS Consortium.

Muscle Nerve. 2019 Feb;59(2):201-207. doi: 10.1002/mus.26335. Epub 2018 Nov 26.

PMID:
30192007
9.

Inclusion Body Myositis: Update on Pathogenesis and Treatment.

Naddaf E, Barohn RJ, Dimachkie MM.

Neurotherapeutics. 2018 Oct;15(4):995-1005. doi: 10.1007/s13311-018-0658-8. Review.

PMID:
30136253
10.

Toxic myopathies.

Pasnoor M, Barohn RJ, Dimachkie MM.

Curr Opin Neurol. 2018 Oct;31(5):575-582. doi: 10.1097/WCO.0000000000000606.

PMID:
30080718
11.

Congenital Myasthenic Syndromes: a Clinical and Treatment Approach.

Farmakidis C, Pasnoor M, Barohn RJ, Dimachkie MM.

Curr Treat Options Neurol. 2018 Jul 21;20(9):36. doi: 10.1007/s11940-018-0520-7. Review.

PMID:
30032336
12.

Update on Inclusion Body Myositis.

Jabari D, Vedanarayanan VV, Barohn RJ, Dimachkie MM.

Curr Rheumatol Rep. 2018 Jun 28;20(8):52. doi: 10.1007/s11926-018-0755-z. Review.

PMID:
29955981
13.

A checklist for clinical trials in rare disease: obstacles and anticipatory actions-lessons learned from the FOR-DMD trial.

Crow RA, Hart KA, McDermott MP, Tawil R, Martens WB, Herr BE, McColl E, Wilkinson J, Kirschner J, King WM, Eagle M, Brown MW, Hirtz D, Lochmuller H, Straub V, Ciafaloni E, Shieh PB, Spinty S, Childs AM, Manzur AY, Morandi L, Butterfield RJ, Horrocks I, Roper H, Flanigan KM, Kuntz NL, Mah JK, Morrison L, Darras BT, von der Hagen M, Schara U, Wilichowski E, Mongini T, McDonald CM, Vita G, Barohn RJ, Finkel RS, Wicklund M, McMillan HJ Jr, Hughes I, Pegoraro E, Bryan Burnette W, Howard JF, Thangarajh M, Campbell C, Griggs RC, Bushby K, Guglieri M.

Trials. 2018 May 10;19(1):291. doi: 10.1186/s13063-018-2645-0. Review.

14.

Fifty Key Publications on Myasthenia Gravis and Related Disorders.

Dimachkie MM, Barohn RJ.

Neurol Clin. 2018 May;36(2):xiii-xvii. doi: 10.1016/j.ncl.2018.03.001. No abstract available.

PMID:
29655457
15.

Lambert-Eaton Myasthenic Syndrome.

Kesner VG, Oh SJ, Dimachkie MM, Barohn RJ.

Neurol Clin. 2018 May;36(2):379-394. doi: 10.1016/j.ncl.2018.01.008. Review.

PMID:
29655456
16.

Measuring Clinical Treatment Response in Myasthenia Gravis.

Barnett C, Herbelin L, Dimachkie MM, Barohn RJ.

Neurol Clin. 2018 May;36(2):339-353. doi: 10.1016/j.ncl.2018.01.006. Review.

PMID:
29655453
17.

Treatment of Myasthenia Gravis.

Farmakidis C, Pasnoor M, Dimachkie MM, Barohn RJ.

Neurol Clin. 2018 May;36(2):311-337. doi: 10.1016/j.ncl.2018.01.011. Review.

PMID:
29655452
18.

Muscle-Specific Tyrosine Kinase and Myasthenia Gravis Owing to Other Antibodies.

Rivner MH, Pasnoor M, Dimachkie MM, Barohn RJ, Mei L.

Neurol Clin. 2018 May;36(2):293-310. doi: 10.1016/j.ncl.2018.01.004. Review.

PMID:
29655451
19.

Diagnosis of Myasthenia Gravis.

Pasnoor M, Dimachkie MM, Farmakidis C, Barohn RJ.

Neurol Clin. 2018 May;36(2):261-274. doi: 10.1016/j.ncl.2018.01.010. Review.

PMID:
29655449
20.

EULAR/ACR classification criteria for adult and juvenile idiopathic inflammatory myopathies and their major subgroups: a methodology report.

Bottai M, Tjärnlund A, Santoni G, Werth VP, Pilkington C, de Visser M, Alfredsson L, Amato AA, Barohn RJ, Liang MH, Singh JA, Aggarwal R, Arnardottir S, Chinoy H, Cooper RG, Danko K, Dimachkie MM, Feldman BM, García-De La Torre I, Gordon P, Hayashi T, Katz JD, Kohsaka H, Lachenbruch PA, Lang BA, Li Y, Oddis CV, Olesinka M, Reed AM, Rutkowska-Sak L, Sanner H, Selva-O'Callaghan A, Wook Song Y, Vencovsky J, Ytterberg SR, Miller FW, Rider LG, Lundberg IE; International Myositis Classification Criteria Project consortium, the Euromyositis register and the Juvenile Dermatomyositis Cohort Biomarker Study and Repository (JDRG) (UK and Ireland) .

RMD Open. 2017 Nov 14;3(2):e000507. doi: 10.1136/rmdopen-2017-000507. eCollection 2017.

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