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Orphanet Journal of Rare Diseases|June 14, 2020
Effects of nusinersen after one year of treatment in 123 children with SMA type 1 or 2: a French real-life observational studyFrédérique Audic, Marta Gomez Garcia de la Banda, Delphine Bernoux, et al.
European Journal of Paediatric Neurology : EJPN : Official Journal of the European Paediatric Neurology Society|September 17, 2011
Motor and respiratory heterogeneity in Duchenne patients: implication for clinical trialsVéronique Humbertclaude, Dalil Hamroun, Kamel Bezzou, et al.
Neurology|October 27, 2019
Longitudinal changes in clinical outcome measures in COL6-related dystrophies and LAMA2-related dystrophiesMinal S Jain, Katherine Meilleur, Eunhee Kim, et al.
Annals of Neurology|April 26, 2018
Congenital Titinopathy: Comprehensive characterization and pathogenic insightsEmily C Oates, Kristi J Jones, Sandra Donkervoort, et al.
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