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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.
Neuromuscular Disorders : NMD|November 10, 2019
Nusinersen initiated in infants during the presymptomatic stage of spinal muscular atrophy: Interim efficacy and safety results from the Phase 2 NURTURE studyDarryl C De Vivo, Enrico Bertini, Kathryn J Swoboda, et al.
Neurology|October 17, 2020
Respiratory Trajectories in Type 2 and 3 Spinal Muscular Atrophy in the iSMAC Cohort StudyFederica Trucco, Deborah Ridout, Mariacristina Scoto, 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.
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.
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.
Annals of Neurology|May 14, 2020
GGPS1 Mutations Cause Muscular Dystrophy/Hearing Loss/Ovarian Insufficiency SyndromeA Reghan Foley, Yaqun Zou, James E Dunford, et al.
Journal of Clinical Medicine|March 11, 2023
2-Year Change in Revised Hammersmith Scale Scores in a Large Cohort of Untreated Paediatric Type 2 and 3 SMA ParticipantsGeorgia Stimpson, Danielle Ramsey, Amy Wolfe, et al.
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