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Neuromuscular Disorders : NMD|March 28, 2021
The Spinal Muscular Atrophy Health Index: Italian validation of a disease-specific outcome measureValeria A Sansone, Alice Pirola, Andrea Lizio, et al.The Lancet. Neurology|February 21, 2023
Antisense oligonucleotide targeting DMPK in patients with myotonic dystrophy type 1: a multicentre, randomised, dose-escalation, placebo-controlled, phase 1/2a trialCharles A Thornton, Richard Thomas Moxley, Katy Eichinger, et al.Muscle & Nerve|May 11, 2026
Longitudinal Psychometric Properties of the Myotonic Dystrophy Health Index in a Large Multicenter Cohort of People Living With Myotonic Dystrophy Type 1Valeria A Sansone, Andrea Lizio, Carola R Ferrari Aggradi, et al.Neurology. Clinical Practice|December 28, 2018
Consensus-based care recommendations for adults with myotonic dystrophy type 1Tetsuo Ashizawa, Cynthia Gagnon, William J Groh, et al.Pageof 7