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JAMA Network Open|January 25, 2022
Efficacy and Safety of Vamorolone in Duchenne Muscular Dystrophy: A 30-Month Nonrandomized Controlled Open-Label Extension TrialJean K Mah, Paula R Clemens, Michela Guglieri, et al.Genetics in Medicine : Official Journal of the American College of Medical Genetics|March 4, 2016
A prospective evaluation of whole-exome sequencing as a first-tier molecular test in infants with suspected monogenic disordersZornitza Stark, Tiong Y Tan, Belinda Chong, et al.Epilepsia|January 21, 2021
The severe epilepsy syndromes of infancy: A population-based studyKatherine B Howell, Jeremy L Freeman, Mark T Mackay, et al.Neurology|August 28, 2019
Vamorolone trial in Duchenne muscular dystrophy shows dose-related improvement of muscle functionEric P Hoffman, Benjamin D Schwartz, Laurel J Mengle-Gaw, et al.Annals of Clinical and Translational Neurology|March 28, 2024
Genome and RNA sequencing boost neuromuscular diagnoses to 62% from 34% with exome sequencing aloneRhett G Marchant, Samantha J Bryen, Melanie Bahlo, et al.Muscle & Nerve|July 22, 2014
Ataluren treatment of patients with nonsense mutation dystrophinopathyKatharine Bushby, Richard Finkel, Brenda Wong, et al.Journal of Comparative Effectiveness Research|August 28, 2020
Meta-analyses of ataluren randomized controlled trials in nonsense mutation Duchenne muscular dystrophyCraig Campbell, Richard J Barohn, Enrico Bertini, et al.Pharmacological Research|September 17, 2018
Phase IIa trial in Duchenne muscular dystrophy shows vamorolone is a first-in-class dissociative steroidal anti-inflammatory drugLaurie S Conklin, Jesse M Damsker, Eric P Hoffman, et al.Brain : a Journal of Neurology|May 1, 2010
Tyrosine hydroxylase deficiency: a treatable disorder of brain catecholamine biosynthesisMichèl A Willemsen, Marcel M Verbeek, Erik-Jan Kamsteeg, et al.Brain Communications|July 9, 2021
International retrospective natural history study of <i>LMNA</i>-related congenital muscular dystrophyRabah Ben Yaou, Pomi Yun, Ivana Dabaj, et al.Pageof 16