Overview of viltolarsen (NS-065/NCNP-01) clinical trials
Trial publications Phase I open-label trial (NCT02081625) Exploratory Study of NS-065/NCNP-01 in DMD Komaki H, Nagata T, Saito T, et…
Overview of drisapersen (GSK2402968/PRO051) clinical trials
Development (and thereby all open-label extension studies) has been terminated Trial publications Exploratory study on the local dystrophin production restoring effect…
Overview of eteplirsen (AVI-4658/Exondys51) clinical trials
Trial publications Phase I local treatment (NCT00159250) Safety and Efficacy Study of Antisense Oligonucleotides in Duchenne Muscular Dystrophy • Kinali M,…
The Effectiveness and Value of Deflazacort and Exon-Skipping Therapies for the Management of Duchenne Muscular Dystrophy
Agboola F, Lin GA, Fluetsch N, Walton SM, Rind DM, Pearson SD. The Effectiveness and Value of Deflazacort and Exon-Skipping…
The administration of antisense oligonucleotide golodirsen reduces pathological regeneration in patients with Duchenne muscular dystrophy
Scaglioni D, Catapano F, Ellis M, et al. The administration of antisense oligonucleotide golodirsen reduces pathological regeneration in patients with…
Restorative treatments of dystrophin expression in Duchenne muscular dystrophy: A systematic review
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…
Gene specific therapies - the next therapeutic milestone in neurology
Brenner D, Ludolph AC, Weishaupt JH. Gene specific therapies - the next therapeutic milestone in neurology. Neurol Res Pract 2020;2:25…
Viltolarsen in Japanese Duchenne muscular dystrophy patients: A phase 1/2 study
Komaki H, Takeshima Y, Matsumura T, et al. Viltolarsen in Japanese Duchenne muscular dystrophy patients: A phase 1/2 study. Annals…
Genotype-Phenotype Correlations in Duchenne and Becker Muscular Dystrophy Patients from the Canadian Neuromuscular Disease Registry
Lim KRQ, Nguyen Q, Yokota T. Genotype-Phenotype Correlations in Duchenne and Becker Muscular Dystrophy Patients from the Canadian Neuromuscular Disease…
Developments in reading frame restoring therapy approaches for Duchenne muscular dystrophy
Schneider AE, Aartsma-Rus A. Developments in reading frame restoring therapy approaches for Duchenne muscular dystrophy. Expert Opin Biol Ther 2020:1-17…
Medical management of muscle weakness in Duchenne muscular dystrophy
Rivera SR, Jhamb SK, Abdel-Hamid HZ, et al. Medical management of muscle weakness in Duchenne muscular dystrophy. Plos One 2020;15:e0240687…
Characterizing Exon Skipping Efficiency in DMD Patient Samples in Clinical Trials of Antisense Oligonucleotides
Nordin JZ, Mizobe Y, Nakamura H, Komaki H, Takeda S, Aoki Y. Characterizing Exon Skipping Efficiency in DMD Patient Samples…
Advances in Genetic Characterization and Genotype-Phenotype Correlation of Duchenne and Becker Muscular Dystrophy in the Personalized Medicine Era
Sheikh O, Yokota T. Advances in Genetic Characterization and Genotype-Phenotype Correlation of Duchenne and Becker Muscular Dystrophy in the Personalized…
