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Antisense oligonucleotide targeting DMPK in patients with myotonic dystrophy type 1: a multicentre, randomised, dose-escalation, placebo-controlled, phase 1/2a trial.
Lancet Neurol. 2023 Mar;22(3):218-228. doi: 10.1016/S1474-4422(23)00001-7.
Lancet Neurol. 2023.
PMID: 36804094
Clinical Trial.
Disease burden and functional outcomes in congenital myotonic dystrophy: A cross-sectional study.
Johnson NE, Butterfield R, Berggren K, Hung M, Chen W, DiBella D, Dixon M, Hayes H, Pucillo E, Bounsanga J, Heatwole C, Campbell C.
Johnson NE, et al.
Neurology. 2016 Jul 12;87(2):160-7. doi: 10.1212/WNL.0000000000002845. Epub 2016 Jun 15.
Neurology. 2016.
PMID: 27306634
Free PMC article.
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Modified dynamic gait index and limits of stability in myotonic dystrophy type 1.
Pucillo EM, Mcintyre MM, Pautler M, Hung M, Bounsanga J, Voss MW, Hayes H, Dibella DL, Trujillo C, Dixon M, Butterfield RJ, Johnson NE.
Pucillo EM, et al.
Muscle Nerve. 2018 Nov;58(5):694-699. doi: 10.1002/mus.26331. Epub 2018 Sep 23.
Muscle Nerve. 2018.
PMID: 30160307
Item in Clipboard
Management of Charcot-Marie-Tooth disease: improving long-term care with a multidisciplinary approach.
McCorquodale D, Pucillo EM, Johnson NE.
McCorquodale D, et al. Among authors: pucillo em.
J Multidiscip Healthc. 2016 Jan 19;9:7-19. doi: 10.2147/JMDH.S69979. eCollection 2016.
J Multidiscip Healthc. 2016.
PMID: 26855581
Free PMC article.
Review.
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Physical function and mobility in children with congenital myotonic dystrophy.
Pucillo EM, Dibella DL, Hung M, Bounsanga J, Crockett B, Dixon M, Butterfield RJ, Campbell C, Johnson NE.
Pucillo EM, et al.
Muscle Nerve. 2017 Aug;56(2):224-229. doi: 10.1002/mus.25482. Epub 2017 Feb 13.
Muscle Nerve. 2017.
PMID: 27859360
Free PMC article.
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Same-day physical therapy consults in an outpatient neuromuscular disease physician clinic.
Pucillo EM, Christensen-Mayer N, Poole SD, Whitten DM, Freeman D, Bohe BR, Swensen BR, Smith AG, Johnson NE.
Pucillo EM, et al.
J Multidiscip Healthc. 2016 Oct 3;9:493-497. doi: 10.2147/JMDH.S113045. eCollection 2016.
J Multidiscip Healthc. 2016.
PMID: 27757040
Free PMC article.
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