Stop codon readthrough Archives • World Duchenne

Overview of Ataluren (Translarna/PTC124) clinical trials

Trial publications Phase II open-label trial (NCT00264888) and extension (NCT00759876) Safety and Efficacy Study of PTC124 in Duchenne Muscular Dystrophy Phase…

Therapeutic approach with Ataluren in Duchenne symptomatic carriers with nonsense mutations in dystrophin gene. Results of a 9-month follow-up in a case report

D'Ambrosio P, Orsini C, Nigro V, Politano L. Therapeutic approach with Ataluren in Duchenne symptomatic carriers with nonsense mutations in…

Restoring Dystrophin Expression in Duchenne Muscular Dystrophy: Current Status of Therapeutic Approaches

Shimizu-Motohashi Y, Komaki H, Motohashi N, Takeda S, Yokota T, Aoki Y. Restoring Dystrophin Expression in Duchenne Muscular Dystrophy: Current…

Mutation-Based Therapeutic Strategies for Duchenne Muscular Dystrophy: From Genetic Diagnosis to Therapy

Nakamura A. Mutation-Based Therapeutic Strategies for Duchenne Muscular Dystrophy: From Genetic Diagnosis to Therapy. Journal of personalized medicine 2019;9 …

A mini-review and implementation model for using ataluren to treat nonsense mutation Duchenne muscular dystrophy

Landfeldt E, Sejersen T, Tulinius M. A mini-review and implementation model for using ataluren to treat nonsense mutation Duchenne muscular…

Ataluren Pharmacokinetics in Healthy Japanese and Caucasian Subjects

Kong R, Laskin OL, Kaushik D, et al. Ataluren Pharmacokinetics in Healthy Japanese and Caucasian Subjects. Clinical pharmacology in drug…

Ataluren in patients with nonsense mutation Duchenne muscular dystrophy (ACT DMD): a multicentre, randomised, double-blind, placebo-controlled, phase 3 trial

McDonald CM, Campbell C, Torricelli RE, et al. Ataluren in patients with nonsense mutation Duchenne muscular dystrophy (ACT DMD): a…

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…

Off-Label Use of Ataluren in Four Non-ambulatory Patients With Nonsense Mutation Duchenne Muscular Dystrophy: Effects on Cardiac and Pulmonary Function and Muscle Strength

Ebrahimi-Fakhari D, Dillmann U, Flotats-Bastardas M, et al. Off-Label Use of Ataluren in Four Non-ambulatory Patients With Nonsense Mutation Duchenne…

One-year follow up of three Italian patients with Duchenne muscular dystrophy treated with ataluren: is earlier better?

Ruggiero L, Iodice R, Esposito M, et al. One-year follow up of three Italian patients with Duchenne muscular dystrophy treated…

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Overview of Ataluren (Translarna/PTC124) clinical trials

Therapeutic approach with Ataluren in Duchenne symptomatic carriers with nonsense mutations in dystrophin gene. Results of a 9-month follow-up in a case report

Restoring Dystrophin Expression in Duchenne Muscular Dystrophy: Current Status of Therapeutic Approaches

Mutation-Based Therapeutic Strategies for Duchenne Muscular Dystrophy: From Genetic Diagnosis to Therapy

A mini-review and implementation model for using ataluren to treat nonsense mutation Duchenne muscular dystrophy

Ataluren Pharmacokinetics in Healthy Japanese and Caucasian Subjects

Ataluren in patients with nonsense mutation Duchenne muscular dystrophy (ACT DMD): a multicentre, randomised, double-blind, placebo-controlled, phase 3 trial

Personalized gene and cell therapy for Duchenne Muscular Dystrophy

Off-Label Use of Ataluren in Four Non-ambulatory Patients With Nonsense Mutation Duchenne Muscular Dystrophy: Effects on Cardiac and Pulmonary Function and Muscle Strength

One-year follow up of three Italian patients with Duchenne muscular dystrophy treated with ataluren: is earlier better?

Road to independence: Documentary about young people with NMD

Webinar Recording: Understanding the role of dystrophin in the brain in DMD/BMD

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