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,…
Functional correction in mouse models of muscular dystrophy using exon-skipping tricyclo-DNA oligomers
Goyenvalle A, Griffith G, Babbs A, El Andaloussi S, Ezzat K, Avril A, et al. Functional correction in mouse models…
Clinical Phenotypes of DMD Exon 51 Skip Equivalent Deletions: A Systematic Review
Waldrop MA, Yaou RB, Lucas KK, et al. Clinical Phenotypes of DMD Exon 51 Skip Equivalent Deletions: A Systematic Review.…
Opportunities and challenges for antisense oligonucleotide therapies
Kuijper EC, Bergsma AJ, Pim Pijnappel WWM, Aartsma-Rus A. Opportunities and challenges for antisense oligonucleotide therapies. J Inherit Metab Dis…
Viltolarsen: First Approval
Dhillon S. Viltolarsen: First Approval. Drugs 2020;80:1027-1031 Abstract Viltolarsen (Viltepso((R)) in Japan) is a phosphorodiamidate morpholino antisense oligonucleotide being…
Optimization of antisense-mediated exon skipping for Duchenne muscular dystrophy
Dzierlega K, Yokota T. Optimization of antisense-mediated exon skipping for Duchenne muscular dystrophy. Gene Ther 2020;27:407-416 Abstract Duchenne muscular…
Safety, Tolerability, and Efficacy of Viltolarsen in Boys With Duchenne Muscular Dystrophy Amenable to Exon 53 Skipping: A Phase 2 Randomized Clinical Trial
Clemens PR, Rao VK, Connolly AM, et al. Safety, Tolerability, and Efficacy of Viltolarsen in Boys With Duchenne Muscular Dystrophy…
Increased dystrophin production with golodirsen in patients with Duchenne muscular dystrophy
Frank DE, Schnell FJ, Akana C, et al. Increased dystrophin production with golodirsen in patients with Duchenne muscular dystrophy. Neurology…
Drisapersen associated with elevated serum factor VIII levels in Duchenne muscular dystrophy
McMillan HJ, Amid A, Gonorazky H, Almobarak S, Campbell C. Drisapersen associated with elevated serum factor VIII levels in Duchenne…
The 10th Oligonucleotide Therapy Approved: Golodirsen for Duchenne Muscular Dystrophy
Aartsma-Rus A, Corey DR. The 10th Oligonucleotide Therapy Approved: Golodirsen for Duchenne Muscular Dystrophy. Nucleic acid therapeutics 2020;30:67-70 Introduction…
The Pharmacokinetics of 2'-O-Methyl Phosphorothioate Antisense Oligonucleotides: Experiences from Developing Exon Skipping Therapies for Duchenne Muscular Dystrophy
Bosgra S, Sipkens J, de Kimpe S, den Besten C, Datson N, van Deutekom J. The Pharmacokinetics of 2'-O-Methyl Phosphorothioate…
