Library

Trial publications Phase I open-label trial (NCT02081625) Exploratory Study of NS-065/NCNP-01 in DMD Komaki H, Nagata T, Saito T, et…

Development (and thereby all open-label extension studies) has been terminated Trial publications Exploratory study on the local dystrophin production restoring effect…

Trial publications Phase I local treatment (NCT00159250) Safety and Efficacy Study of Antisense Oligonucleotides in Duchenne Muscular Dystrophy • Kinali M,…

Waldrop MA, Yaou RB, Lucas KK, et al. Clinical Phenotypes of DMD Exon 51 Skip Equivalent Deletions: A Systematic Review.…

Kuijper EC, Bergsma AJ, Pim Pijnappel WWM, Aartsma-Rus A. Opportunities and challenges for antisense oligonucleotide therapies. J Inherit Metab Dis…

Dhillon S. Viltolarsen: First Approval. Drugs 2020;80:1027-1031   Abstract Viltolarsen (Viltepso((R)) in Japan) is a phosphorodiamidate morpholino antisense oligonucleotide being…

Dzierlega K, Yokota T. Optimization of antisense-mediated exon skipping for Duchenne muscular dystrophy. Gene Ther 2020   Abstract Duchenne muscular…

Clemens PR, Rao VK, Connolly AM, et al. Safety, Tolerability, and Efficacy of Viltolarsen in Boys With Duchenne Muscular Dystrophy…

Frank DE, Schnell FJ, Akana C, et al. Increased dystrophin production with golodirsen in patients with Duchenne muscular dystrophy. Neurology…

McMillan HJ, Amid A, Gonorazky H, Almobarak S, Campbell C. Drisapersen associated with elevated serum factor VIII levels in Duchenne…

Aartsma-Rus A, Corey DR. The 10th Oligonucleotide Therapy Approved: Golodirsen for Duchenne Muscular Dystrophy. Nucleic acid therapeutics 2020;30:67-70   Introduction…

Bosgra S, Sipkens J, de Kimpe S, den Besten C, Datson N, van Deutekom J. The Pharmacokinetics of 2'-O-Methyl Phosphorothioate…

Domingos J, Ricotti V, Martinez AE, Muntoni F. Severe persistent injection site reactions after subcutaneous 2'-O-methyl phosphorothioate oligonucleotide therapy for…