PTC Therapeitics Reports Ataluren Clinical Data Demonstrate an Increase in Dystrophin Expression in nmDMD Patients

PTC Therapeutics PTCT today announced the publication of data in PLOS ONE demonstrating that nonsense mutation Duchenne muscular dystrophy (nmDMD) patients treated with ataluren, an investigational new drug, experienced an increase in dystrophin expression. These data were obtained from PTC's Phase 2a open-label trial of ataluren in which change in full-length dystrophin expression, as assessed by immunofluorescent staining, was the primary endpoint. Patients with nmDMD typically experience decreased levels of dystrophin protein, which is critical to normal muscle function. The study evaluated whether ataluren restores dystrophin production in the muscle cells of patients with nmDMD. Excisions of the extensor digitorum brevis (EDB) muscles were performed pre- and post-treatment with ataluren on all subjects, and immunofluorescence images were analyzed qualitatively and quantitatively. "The results of this Phase 2a study support ataluren's activity as a dystrophin restoration therapy in nonsense mutation Duchenne muscular dystrophy patients mediated by ribosomal read-through of nonsense mutations," said Stuart Peltz, Ph.D., CEO of PTC Therapeutics, Inc. "These positive findings were the basis for the progression into a Phase 2b study of ataluren in patients with nmDMD, which further demonstrated the potential See full press release