admin September 10, 2017

Ataluren is conditionally approved by the European medicines agency to treat nonsense mutation Duchenne muscular dystrophy (nmDMD), with a specific obligation to conduct a long-term trial. This phase 3, randomized, double-blind, placebo-controlled trial with an open-label extension period is designed to evaluate the long-term efficacy and safety of ataluren in boys with nmDMD (Study 041). Patients eligible for participation will include those with phenotypic evidence of DMD, a nonsense mutation in the dystrophin gene, age ≥ 5 y, corticosteroid use for at least 12 months, 6-minute walk distance (6MWD) ≥ 150 m, and ability to perform timed function tests (TFTs) within 30 s.

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