Library

Trial publications Phase I open-label trial (NCT02081625) Exploratory Study of NS-065/NCNP-01 in DMD Komaki H, Nagata T, Saito T, et…

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,…

Agboola F, Lin GA, Fluetsch N, Walton SM, Rind DM, Pearson SD. The Effectiveness and Value of Deflazacort and Exon-Skipping…

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…

Brenner D, Ludolph AC, Weishaupt JH. Gene specific therapies - the next therapeutic milestone in neurology. Neurol Res Pract 2020;2:25…

Komaki H, Takeshima Y, Matsumura T, et al. Viltolarsen in Japanese Duchenne muscular dystrophy patients: A phase 1/2 study. Annals…

Lim KRQ, Nguyen Q, Yokota T. Genotype-Phenotype Correlations in Duchenne and Becker Muscular Dystrophy Patients from the Canadian Neuromuscular Disease…

Schneider AE, Aartsma-Rus A. Developments in reading frame restoring therapy approaches for Duchenne muscular dystrophy. Expert Opin Biol Ther 2020:1-17…

Rivera SR, Jhamb SK, Abdel-Hamid HZ, et al. Medical management of muscle weakness in Duchenne muscular dystrophy. Plos One 2020;15:e0240687…

Nordin JZ, Mizobe Y, Nakamura H, Komaki H, Takeda S, Aoki Y. Characterizing Exon Skipping Efficiency in DMD Patient Samples…

Sheikh O, Yokota T. Advances in Genetic Characterization and Genotype-Phenotype Correlation of Duchenne and Becker Muscular Dystrophy in the Personalized…