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,…
Systematic evaluation of 2'-Fluoro modified chimeric antisense oligonucleotide-mediated exon skipping in vitro
Chen S, Le BT, Chakravarthy M, Kosbar TR, Veedu RN. Systematic evaluation of 2'-Fluoro modified chimeric antisense oligonucleotide-mediated exon skipping…
Eteplirsen Treatment Attenuates Respiratory Decline in Ambulatory and Non Ambulatory Patients with Duchenne Muscular Dystrophy
Khan N, Eliopoulos H, Han L, et al. Eteplirsen Treatment Attenuates Respiratory Decline in Ambulatory and Non Ambulatory Patients with…
Restoring Dystrophin Expression in Duchenne Muscular Dystrophy: Current Status of Therapeutic Approaches
Shimizu-Motohashi Y, Komaki H, Motohashi N, Takeda S, Yokota T, Aoki Y. Restoring Dystrophin Expression in Duchenne Muscular Dystrophy: Current…
Mutation-Based Therapy for Duchenne Muscular Dystrophy: Antisense Treatment Arrives in the Clinic
McNally EM, Wyatt EJ. Mutation-Based Therapy for Duchenne Muscular Dystrophy: Antisense Treatment Arrives in the Clinic. Circulation 2017;136:979-981
Mutation-Based Therapeutic Strategies for Duchenne Muscular Dystrophy: From Genetic Diagnosis to Therapy
Nakamura A. Mutation-Based Therapeutic Strategies for Duchenne Muscular Dystrophy: From Genetic Diagnosis to Therapy. Journal of personalized medicine 2019;9 …
Nonclinical Exon Skipping Studies with 2'-O-Methyl Phosphorothioate Antisense Oligonucleotides in mdx and mdx-utrn-/- Mice Inspired by Clinical Trial Results
van Putten M, Tanganyika-de Winter C, Bosgra S, Aartsma-Rus A. Nonclinical Exon Skipping Studies with 2'-O-Methyl Phosphorothioate Antisense Oligonucleotides in…
Placebo-controlled Phase 2 Trial of Drisapersen for Duchenne Muscular Dystrophy
McDonald CM, Wong B, Flanigan KM, et al. Placebo-controlled Phase 2 Trial of Drisapersen for Duchenne Muscular Dystrophy. Annals of…
DMD genotype correlations from the Duchenne Registry: Endogenous exon skipping is a factor in prolonged ambulation for individuals with a defined mutation subtype
Wang RT, Barthelemy F, Martin AS, et al. DMD genotype correlations from the Duchenne Registry: Endogenous exon skipping is a…
Exon-skipping advances for Duchenne muscular dystrophy
Echevarria L, Aupy P, Goyenvalle A. Exon-skipping advances for Duchenne muscular dystrophy. Human molecular genetics 2018;27:R163-R172 Abstract Duchenne muscular…
Eteplirsen treatment for Duchenne muscular dystrophy: Exon skipping and dystrophin production
Charleston JS, Schnell FJ, Dworzak J, et al. Eteplirsen treatment for Duchenne muscular dystrophy: Exon skipping and dystrophin production. Neurology…
