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Bioorganic & Medicinal Chemistry|December 12, 2018
Synthesis and evaluation of esterified Hsp70 agonists in cellular models of protein aggregation and foldingAnnette N Chiang, Mary Liang, Antonio Dominguez-Meijide, et al.Neuromuscular Disorders : NMD|October 30, 2016
Developmental milestones in type I spinal muscular atrophyRoberto De Sanctis, Giorgia Coratti, Amy Pasternak, et al.Sleep Medicine|February 21, 2017
Sleep disorders in spinal muscular atrophyMaria C Pera, Domenico M Romeo, Alessandra Graziano, et al.Plos One|April 26, 2023
Determining minimal clinically important differences in the North Star Ambulatory Assessment (NSAA) for patients with Duchenne muscular dystrophyVandana Ayyar Gupta, Jacqueline M Pitchforth, Joana Domingos, et al.Muscle & Nerve|April 8, 2015
Spinal muscular atrophy functional composite score: A functional measure in spinal muscular atrophyJacqueline Montes, Allan M Glanzman, Elena S Mazzone, et al.European Journal of Neurology|July 15, 2022
Risdiplam in types 2 and 3 spinal muscular atrophy: A randomised, placebo-controlled, dose-finding trial followed by 24 months of treatmentEugenio Mercuri, Giovanni Baranello, Odile Boespflug-Tanguy, et al.Journal of Neuromuscular Diseases|June 6, 2018
Evaluator Training and Reliability for SMA Global Nusinersen Trials1Allan M Glanzman, Elena S Mazzone, Sally Dunaway Young, et al.Journal of Neurology|February 3, 2023
Two-year efficacy and safety of risdiplam in patients with type 2 or non-ambulant type 3 spinal muscular atrophy (SMA)Maryam Oskoui, John W Day, Nicolas Deconinck, et al.The Lancet. Neurology|December 23, 2021
Safety and efficacy of once-daily risdiplam in type 2 and non-ambulant type 3 spinal muscular atrophy (SUNFISH part 2): a phase 3, double-blind, randomised, placebo-controlled trialEugenio Mercuri, Nicolas Deconinck, Elena S Mazzone, et al.Neuromuscular Disorders : NMD|January 2, 2018
Diagnosis and management of spinal muscular atrophy: Part 1: Recommendations for diagnosis, rehabilitation, orthopedic and nutritional careEugenio Mercuri, Richard S Finkel, Francesco Muntoni, et al.Pageof 8