Long-Term Safety and Efficacy Data of Golodirsen in Ambulatory Patients with Duchenne Muscular Dystrophy Amenable to Exon 53 Skipping: A First-in-human, Multicenter, Two-Part, Open-Label, Phase 1/2 TrialLaurent Servais, Frederick J. Schnell, Andreea Seferian et al.|Nucleic Acid Therapeutics|2021Cited by 128
Titin copy number variations associated with dominant inherited phenotypesAurélien Perrin, Mireille Cossée, Corinne Métay et al.|Journal of Medical Genetics|2023Cited by 5