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Items: 1 to 20 of 170

1.

Current treatment practice of Guillain-Barré syndrome.

Verboon C, Doets AY, Galassi G, Davidson A, Waheed W, Péréon Y, Shahrizaila N, Kusunoki S, Lehmann HC, Harbo T, Monges S, Van den Bergh P, Willison HJ, Cornblath DR, Jacobs BC; IGOS Consortium.

Neurology. 2019 Jul 2;93(1):e59-e76. doi: 10.1212/WNL.0000000000007719. Epub 2019 Jun 7.

PMID:
31175208
2.

Towards regulatory endorsement of drug development tools to promote the application of model-informed drug development in Duchenne muscular dystrophy.

Conrado DJ, Larkindale J, Berg A, Hill M, Burton J, Abrams KR, Abresch RT, Bronson A, Chapman D, Crowther M, Duong T, Gordish-Dressman H, Harnisch L, Henricson E, Kim S, McDonald CM, Schmidt S, Vong C, Wang X, Wong BL, Yong F, Romero K; Duchenne Muscular Dystrophy Regulatory Science Consortium (D-RSC).

J Pharmacokinet Pharmacodyn. 2019 May 24. doi: 10.1007/s10928-019-09642-7. [Epub ahead of print]

PMID:
31127458
3.

Correction to: Eculizumab improves fatigue in refractory generalized myasthenia gravis.

Andersen H, Mantegazza R, Wang JJ, O'Brien F, Patra K, Howard JF Jr; REGAIN Study Group.

Qual Life Res. 2019 Aug;28(8):2255. doi: 10.1007/s11136-019-02204-x.

4.

ULK1 and ULK2 Regulate Stress Granule Disassembly Through Phosphorylation and Activation of VCP/p97.

Wang B, Maxwell BA, Joo JH, Gwon Y, Messing J, Mishra A, Shaw TI, Ward AL, Quan H, Sakurada SM, Pruett-Miller SM, Bertorini T, Vogel P, Kim HJ, Peng J, Taylor JP, Kundu M.

Mol Cell. 2019 May 16;74(4):742-757.e8. doi: 10.1016/j.molcel.2019.03.027. Epub 2019 Apr 9.

PMID:
30979586
5.

Eculizumab improves fatigue in refractory generalized myasthenia gravis.

Andersen H, Mantegazza R, Wang JJ, O'Brien F, Patra K, Howard JF Jr; REGAIN Study Group.

Qual Life Res. 2019 Aug;28(8):2247-2254. doi: 10.1007/s11136-019-02148-2. Epub 2019 Mar 23. Erratum in: Qual Life Res. 2019 May 21;:.

6.

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 Jul;60(1):14-24. doi: 10.1002/mus.26447. Epub 2019 Mar 8.

7.

Longitudinal pulmonary function testing outcome measures in Duchenne muscular dystrophy: Long-term natural history with and without glucocorticoids.

McDonald CM, Gordish-Dressman H, Henricson EK, Duong T, Joyce NC, Jhawar S, Leinonen M, Hsu F, Connolly AM, Cnaan A, Abresch RT; CINRG investigators for PubMed.

Neuromuscul Disord. 2018 Nov;28(11):897-909. doi: 10.1016/j.nmd.2018.07.004. Epub 2018 Aug 29.

8.

Regional variation of Guillain-Barré syndrome.

Doets AY, Verboon C, van den Berg B, Harbo T, Cornblath DR, Willison HJ, Islam Z, Attarian S, Barroso FA, Bateman K, Benedetti L, van den Bergh P, Casasnovas C, Cavaletti G, Chavada G, Claeys KG, Dardiotis E, Davidson A, van Doorn PA, Feasby TE, Galassi G, Gorson KC, Hartung HP, Hsieh ST, Hughes RAC, Illa I, Islam B, Kusunoki S, Kuwabara S, Lehmann HC, Miller JAL, Mohammad QD, Monges S, Nobile Orazio E, Pardo J, Pereon Y, Rinaldi S, Querol L, Reddel SW, Reisin RC, Shahrizaila N, Sindrup SH, Waqar W, Jacobs BC; IGOS Consortium.

Brain. 2018 Oct 1;141(10):2866-2877. doi: 10.1093/brain/awy232.

9.

Safety and efficacy of eculizumab in anti-acetylcholine receptor antibody-positive refractory generalised myasthenia gravis (REGAIN): a phase 3, randomised, double-blind, placebo-controlled, multicentre study.

Howard JF Jr, Utsugisawa K, Benatar M, Murai H, Barohn RJ, Illa I, Jacob S, Vissing J, Burns TM, Kissel JT, Muppidi S, Nowak RJ, O'Brien F, Wang JJ, Mantegazza R; REGAIN Study Group.

Lancet Neurol. 2017 Dec;16(12):976-986. doi: 10.1016/S1474-4422(17)30369-1. Epub 2017 Oct 20. Erratum in: Lancet Neurol. 2017 Dec;16(12 ):954.

PMID:
29066163
10.

Interleukin 1 Receptor-Like 1 Protein (ST2) is a Potential Biomarker for Cardiomyopathy in Duchenne Muscular Dystrophy.

Anderson J, Seol H, Gordish-Dressman H, Hathout Y, Spurney CF; CINRG Investigators.

Pediatr Cardiol. 2017 Dec;38(8):1606-1612. doi: 10.1007/s00246-017-1703-9. Epub 2017 Aug 18.

11.

A Man With Distal Asymmetric Leg Weakness.

Bello R, Bertorini T.

J Clin Neuromuscul Dis. 2017 Jun;18(4):235-243. doi: 10.1097/CND.0000000000000147.

PMID:
28538255
12.

International Guillain-Barré Syndrome Outcome Study: protocol of a prospective observational cohort study on clinical and biological predictors of disease course and outcome in Guillain-Barré syndrome.

Jacobs BC, van den Berg B, Verboon C, Chavada G, Cornblath DR, Gorson KC, Harbo T, Hartung HP, Hughes RAC, Kusunoki S, van Doorn PA, Willison HJ; IGOS Consortium.

J Peripher Nerv Syst. 2017 Jun;22(2):68-76. doi: 10.1111/jns.12209.

PMID:
28406555
13.

A Case of Asymptomatic Inclusion Body Myositis.

Bello R, Bertorini T, Ganta K, Mays W.

J Clin Neuromuscul Dis. 2017 Mar;18(3):132-134. doi: 10.1097/CND.0000000000000142.

PMID:
28221303
14.

Evidence for ACTN3 as a genetic modifier of Duchenne muscular dystrophy.

Hogarth MW, Houweling PJ, Thomas KC, Gordish-Dressman H, Bello L; Cooperative International Neuromuscular Research Group (CINRG), Pegoraro E, Hoffman EP, Head SI, North KN.

Nat Commun. 2017 Jan 31;8:14143. doi: 10.1038/ncomms14143.

15.

Targeted Re-Sequencing Emulsion PCR Panel for Myopathies: Results in 94 Cases.

Punetha J, Kesari A, Uapinyoying P, Giri M, Clarke NF, Waddell LB, North KN, Ghaoui R, O'Grady GL, Oates EC, Sandaradura SA, Bönnemann CG, Donkervoort S, Plotz PH, Smith EC, Tesi-Rocha C, Bertorini TE, Tarnopolsky MA, Reitter B, Hausmanowa-Petrusewicz I, Hoffman EP.

J Neuromuscul Dis. 2016 May 27;3(2):209-225.

PMID:
27854218
16.

Association Study of Exon Variants in the NF-κB and TGFβ Pathways Identifies CD40 as a Modifier of Duchenne Muscular Dystrophy.

Bello L, Flanigan KM, Weiss RB; United Dystrophinopathy Project, Spitali P, Aartsma-Rus A, Muntoni F, Zaharieva I, Ferlini A, Mercuri E, Tuffery-Giraud S, Claustres M, Straub V, Lochmüller H, Barp A, Vianello S, Pegoraro E, Punetha J, Gordish-Dressman H, Giri M, McDonald CM, Hoffman EP; Cooperative International Neuromuscular Research Group.

Am J Hum Genet. 2016 Nov 3;99(5):1163-1171. doi: 10.1016/j.ajhg.2016.08.023. Epub 2016 Oct 13.

17.

Comparative Long-Term Evaluation of Patients With Juvenile Inflammatory Myopathies.

Elahi HA, Bertorini TE, Igarashi M, Mays WH 3rd, Whitaker JN.

J Clin Neuromuscul Dis. 2016 Sep;18(1):21-7. doi: 10.1097/CND.0000000000000131.

PMID:
27552385
18.

DMD genotypes and loss of ambulation in the CINRG Duchenne Natural History Study.

Bello L, Morgenroth LP, Gordish-Dressman H, Hoffman EP, McDonald CM, Cirak S; CINRG investigators.

Neurology. 2016 Jul 26;87(4):401-9. doi: 10.1212/WNL.0000000000002891. Epub 2016 Jun 24.

19.

Somatic mosaicism due to a reversion variant causing hemi-atrophy: a novel variant of dystrophinopathy.

Punetha J, Mansoor S, Bertorini TE, Kesari A, Brown KJ, Hoffman EP.

Eur J Hum Genet. 2016 Oct;24(10):1511-4. doi: 10.1038/ejhg.2016.22. Epub 2016 Mar 9.

20.

An Overview of Laryngeal Muscle Single Fiber Electromyography.

Bertorini TE, Sharaf AG.

J Clin Neurophysiol. 2015 Aug;32(4):304-8. doi: 10.1097/WNP.0000000000000176. Review.

PMID:
26241239

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