Hirofumi Komaki

TL;DR: It is suggested that drisapersen could have benefit in a less impaired population of DMD subjects and the statistical power from pre-specified 90% to actual 53%.

TL;DR: A phase 1 clinical trial of NS-065/NCNP-01 showed a favorable safety profile and pharmacokinetics, and the authors demonstrated that it effectively skipped exon 53 in the dystrophin gene, suggesting that a phase 2 trial of the drug is warranted.

TL;DR: STIR is sufficient to assist clinicians in diagnosing CIDP and T1-weighted images and DWIs seemed useful for speculating about the pathological changes in swollen plexuses in C IDP patients.

TL;DR: In this paper, exon skipping that targets exons 51, 44, 45, and 53 is being globally investigated including in USA, EU, and Japan, demonstrating successful dystrophin production in muscles of patients with Duchenne muscular dystrophy after treating with exon 53 skipping antisense oligonucleotides (ASOs).

TL;DR: Levels of miR-1, mi-133a, and mi-206 in serum of BMD and miR -1 in sera of LGMD and FSHD patients showed no significant differences compared with those of controls by Bonferroni correction, but results might need increase in sample sizes to evaluate these three miRNAs as variable biomarkers.