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,…
Longitudinal effect of eteplirsen versus historical control on ambulation in Duchenne muscular dystrophy
Mendell JR, Goemans N, Lowes LP, et al. Longitudinal effect of eteplirsen versus historical control on ambulation in Duchenne muscular…
Eteplirsen for the treatment of Duchenne muscular dystrophy
Mendell JR, Rodino-Klapac LR, Sahenk Z, et al. Eteplirsen for the treatment of Duchenne muscular dystrophy. Annals of neurology 2013;74:637-47…
FDA Approves Eteplirsen for Duchenne Muscular Dystrophy: The Next Chapter in the Eteplirsen Saga
Aartsma-Rus A, Krieg AM. FDA Approves Eteplirsen for Duchenne Muscular Dystrophy: The Next Chapter in the Eteplirsen Saga. Nucleic acid…
Systemic administration of the antisense oligonucleotide NS-065/NCNP-01 for skipping of exon 53 in patients with Duchenne muscular dystrophy
Komaki H, Nagata T, Saito T, et al. Systemic administration of the antisense oligonucleotide NS-065/NCNP-01 for skipping of exon 53…
Personalized gene and cell therapy for Duchenne Muscular Dystrophy
Barthelemy F, Wein N. Personalized gene and cell therapy for Duchenne Muscular Dystrophy. Neuromuscular disorders : NMD 2018;28:803-824 Abstract…
NS-065/NCNP-01: An Antisense Oligonucleotide for Potential Treatment of Exon 53 Skipping in Duchenne Muscular Dystrophy
Watanabe N, Nagata T, Satou Y, et al. NS-065/NCNP-01: An Antisense Oligonucleotide for Potential Treatment of Exon 53 Skipping in…
Injection site reactions after long-term subcutaneous delivery of drisapersen: a retrospective study
Hilhorst N, Spanoudi-Kitrimi I, Goemans N, Morren MA. Injection site reactions after long-term subcutaneous delivery of drisapersen: a retrospective study.…
Multiple Exon Skipping in the Duchenne Muscular Dystrophy Hot Spots: Prospects and Challenges
Echigoya Y, Lim KRQ, Nakamura A, Yokota T. Multiple Exon Skipping in the Duchenne Muscular Dystrophy Hot Spots: Prospects and…
A Sequel to the Eteplirsen Saga: Eteplirsen Is Approved in the United States but Was Not Approved in Europe
Aartsma-Rus A, Goemans N. A Sequel to the Eteplirsen Saga: Eteplirsen Is Approved in the United States but Was Not…
Translational and Regulatory Challenges for Exon Skipping Therapies
Aartsma-Rus A, Ferlini A, Goemans N, et al. Translational and regulatory challenges for exon skipping therapies. Human gene therapy 2014;25:885-92…
