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,…
Severe persistent injection site reactions after subcutaneous 2'-O-methyl phosphorothioate oligonucleotide therapy for Duchenne muscular dystrophy
Domingos J, Ricotti V, Martinez AE, Muntoni F. Severe persistent injection site reactions after subcutaneous 2'-O-methyl phosphorothioate oligonucleotide therapy for…
Therapy with 2'-O-Me Phosphorothioate Antisense Oligonucleotides Causes Reversible Proteinuria by Inhibiting Renal Protein Reabsorption
Janssen MJ, Nieskens TTG, Steevels TAM, et al. Therapy with 2'-O-Me Phosphorothioate Antisense Oligonucleotides Causes Reversible Proteinuria by Inhibiting Renal…
A randomized placebo-controlled phase 3 trial of an antisense oligonucleotide, drisapersen, in Duchenne muscular dystrophy
Goemans N, Mercuri E, Belousova E, et al. A randomized placebo-controlled phase 3 trial of an antisense oligonucleotide, drisapersen, in…
Safety and efficacy of drisapersen for the treatment of Duchenne muscular dystrophy (DEMAND II): an exploratory, randomised, placebo-controlled phase 2 study
Voit T, Topaloglu H, Straub V, et al. Safety and efficacy of drisapersen for the treatment of Duchenne muscular dystrophy…
Pharmacokinetics and safety of single doses of drisapersen in non-ambulant subjects with Duchenne muscular dystrophy: results of a double-blind randomized clinical trial
Flanigan KM, Voit T, Rosales XQ, et al. Pharmacokinetics and safety of single doses of drisapersen in non-ambulant subjects with…
Comparison of ambulatory capacity and disease progression of Duchenne muscular dystrophy subjects enrolled in the drisapersen DMD114673 study with a matched natural history cohort of subjects on daily corticosteroids
Goemans N, Tulinius M, Kroksmark AK, Wilson R, van den Hauwe M, Campion G. Comparison of ambulatory capacity and disease…
Long-Term Efficacy, Safety, and Pharmacokinetics of Drisapersen in Duchenne Muscular Dystrophy: Results from an Open-Label Extension Study
Goemans NM, Tulinius M, van den Hauwe M, et al. Long-Term Efficacy, Safety, and Pharmacokinetics of Drisapersen in Duchenne Muscular…
Systemic administration of PRO051 in Duchenne's muscular dystrophy
Goemans NM, Tulinius M, van den Akker JT, et al. Systemic administration of PRO051 in Duchenne's muscular dystrophy. The New…
Local dystrophin restoration with antisense oligonucleotide PRO051
van Deutekom JC, Janson AA, Ginjaar IB, et al. Local dystrophin restoration with antisense oligonucleotide PRO051. The New England journal…
Long-Term Pulmonary Function in Duchenne Muscular Dystrophy: Comparison of Eteplirsen-Treated Patients to Natural History
Kinane TB, Mayer OH, Duda PW, Lowes LP, Moody SL, Mendell JR. Long-Term Pulmonary Function in Duchenne Muscular Dystrophy: Comparison…
