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Neurology|May 9, 2008
Cell-based interventions for neurologic conditions: ethical challenges for early human trialsD J H Mathews, J Sugarman, H Bok, et al.
JAMA Network Open|September 23, 2024
Positive Autism Screening Rates in Toddlers Born During the COVID-19 PandemicMorgan R Firestein, Angela Manessis, Jennifer M Warmingham, et al.
Plos Medicine|September 21, 2020
Efficacy and safety of vamorolone in Duchenne muscular dystrophy: An 18-month interim analysis of a non-randomized open-label extension studyEdward C Smith, Laurie S Conklin, Eric P Hoffman, et al.
Nature Communications|July 2, 2025
Nitrogen and phosphorus differentially control marine biomass production and stoichiometryEmily A Seelen, Samantha J Gleich, William Kumler, et al.
The Lancet. Neurology|August 16, 2025
Safety and efficacy of apitegromab in nonambulatory type 2 or type 3 spinal muscular atrophy (SAPPHIRE): a phase 3, double-blind, randomised, placebo-controlled trialThomas O Crawford, Laurent Servais, Eugenio Mercuri, et al.
Human Mutation|November 26, 2009
Mutational spectrum of DMD mutations in dystrophinopathy patients: application of modern diagnostic techniques to a large cohortKevin M Flanigan, Diane M Dunn, Andrew von Niederhausern, et al.
Neurology|October 19, 2012
Prospective cohort study of spinal muscular atrophy types 2 and 3Petra Kaufmann, Michael P McDermott, Basil T Darras, et al.
European Journal of Neurology|April 24, 2024
Determining minimal clinically important differences in the Hammersmith Functional Motor Scale Expanded for untreated spinal muscular atrophy patients: An international studyGiorgia Coratti, Francesca Bovis, Maria Carmela Pera, et al.
Neuromuscular Disorders : NMD|September 19, 2024
Upper limb function changes over 12 months in untreated SMA II and III individuals: an item-level analysis using the Revised Upper Limb ModuleGiorgia Coratti, Matthew Civitello, Annemarie Rohwer, et al.
Brain : a Journal of Neurology|June 7, 2023
Genetic analysis and natural history of Charcot-Marie-Tooth disease CMTX1 due to GJB1 variantsChristopher J Record, Mariola Skorupinska, Matilde Laura, et al.
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