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The Lancet. Neurology|November 6, 2007
Efficacy of minocycline in patients with amyotrophic lateral sclerosis: a phase III randomised trialPaul H Gordon, Dan H Moore, Robert G Miller, et al.
Neuromuscular Disorders : NMD|February 17, 2019
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 studyLoren D M Pena, Richard J Barohn, Barry J Byrne, et al.
Neurology|May 26, 2022
Long-term Safety and Efficacy of Avalglucosidase Alfa in Patients With Late-Onset Pompe DiseaseMazen M Dimachkie, Richard J Barohn, Barry Byrne, et al.
Annals of Neurology|June 3, 2010
Gentamicin-induced readthrough of stop codons in Duchenne muscular dystrophyVinod Malik, Louise R Rodino-Klapac, Laurence Viollet, et al.
Neurobiology of Aging|February 12, 2015
The effects of an intronic polymorphism in TOMM40 and APOE genotypes in sporadic inclusion body myositisQiang Gang, Conceicao Bettencourt, Pedro M Machado, et al.
Molecular Genetics and Metabolism|July 31, 2016
Prospective exploratory muscle biopsy, imaging, and functional assessment in patients with late-onset Pompe disease treated with alglucosidase alfa: The EMBASSY StudyAns van der Ploeg, Pierre G Carlier, Robert-Yves Carlier, et al.
Neurology|January 24, 2016
Cognitive-behavioral screening reveals prevalent impairment in a large multicenter ALS cohortJennifer Murphy, Pam Factor-Litvak, Raymond Goetz, et al.
Science Translational Medicine|March 25, 2016
Targeting protein homeostasis in sporadic inclusion body myositisMhoriam Ahmed, Pedro M Machado, Adrian Miller, et al.
Annals of Neurology|March 13, 2008
A phase I/IItrial of MYO-029 in adult subjects with muscular dystrophyKathryn R Wagner, James L Fleckenstein, Anthony A Amato, et al.
Neurology|June 17, 2016
A randomized controlled trial of methotrexate for patients with generalized myasthenia gravisMamatha Pasnoor, Jianghua He, Laura Herbelin, et al.
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