Development (and thereby all open-label extension studies) has been terminated Trial publications Exploratory study on the local dystrophin production restoring effect…
Trial publications Phase I local treatment (NCT00159250) Safety and Efficacy Study of Antisense Oligonucleotides in Duchenne Muscular Dystrophy • Kinali M,…
Scaglioni D, Catapano F, Ellis M, et al. The administration of antisense oligonucleotide golodirsen reduces pathological regeneration in patients with…
Pascual-Morena C, Cavero-Redondo I, Álvarez-Bueno C, Mesas AE, Pozuelo-Carrascosa D, Martínez-Vizcaíno V. Restorative treatments of dystrophin expression in Duchenne muscular…
Lim KRQ, Nguyen Q, Yokota T. Genotype-Phenotype Correlations in Duchenne and Becker Muscular Dystrophy Patients from the Canadian Neuromuscular Disease…
Sheikh O, Yokota T. Advances in Genetic Characterization and Genotype-Phenotype Correlation of Duchenne and Becker Muscular Dystrophy in the Personalized…
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