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,…

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…

Janssen MJ, Nieskens TTG, Steevels TAM, et al. Therapy with 2'-O-Me Phosphorothioate Antisense Oligonucleotides Causes Reversible Proteinuria by Inhibiting Renal…

Goemans N, Mercuri E, Belousova E, et al. A randomized placebo-controlled phase 3 trial of an antisense oligonucleotide, drisapersen, in…

Voit T, Topaloglu H, Straub V, et al. Safety and efficacy of drisapersen for the treatment of Duchenne muscular dystrophy…

Flanigan KM, Voit T, Rosales XQ, et al. Pharmacokinetics and safety of single doses of drisapersen in non-ambulant subjects with…