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Neuromuscular Disorders : NMD|September 9, 2020
Age and baseline values predict 12 and 24-month functional changes in type 2 SMAGiorgia Coratti, Maria C Pera, Simona Lucibello, et al.Neurology|February 1, 2023
<i>DMD</i> Genotypes and Motor Function in Duchenne Muscular Dystrophy: A Multi-institution Meta-analysis With Implications for Clinical TrialsFrancesco Muntoni, James Signorovitch, Gautam Sajeev, 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 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.Translational Psychiatry|August 23, 2019
Dystrophin deficiency leads to dysfunctional glutamate clearance in iPSC derived astrocytesAbdulsamie M Patel, Keimpe Wierda, Lieven Thorrez, et al.The Lancet. Neurology|June 16, 2016
Stakeholder cooperation to overcome challenges in orphan medicine development: the example of Duchenne muscular dystrophyVolker Straub, Pavel Balabanov, Kate Bushby, 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.Plos One|August 1, 2019
Correction: Long-term natural history data in Duchenne muscular dystrophy ambulant patients with mutations amenable to skip exons 44, 45, 51 and 53Claudia Brogna, Giorgia Coratt, Marika Pane, et al.Plos One|June 26, 2019
Long-term natural history data in Duchenne muscular dystrophy ambulant patients with mutations amenable to skip exons 44, 45, 51 and 53Claudia Brogna, Giorgia Coratti, Marika Pane, et al.Annals of Neurology|June 29, 2005
CINRG randomized controlled trial of creatine and glutamine in Duchenne muscular dystrophyDiana M Escolar, Gunnar Buyse, Erik Henricson, et al.Pageof 11