Potential of eteplirsen in preserving respiratory function in patients with DMD


Duchenne muscular dystrophy (DMD)  is primarily caused by whole exon deletions, resulting in a shift of the dystrophin mRNA reading frame that prevents production of functional dystrophin protein. Eteplirsen, a phosphorodiamidate morpholino oligomer (PMO), is designed to skip exon 51, restore the reading frame, and induce production of internally shortened dystrophin in patients with mutations amenable to such treatment. This study describes lung function assessed throughout eteplirsen studies 201/202. Study 201 comprised a 24-week, randomized, placebo-controlled period followed by a 4-week open-label period, and study 202 was a 212-week, open-label safety extension study. These consecutive studies assessed the safety and efficacy of eteplirsen. The primary end point was the 6-minute walk test (6MWT), and data on this and other clinical and biomarker end points were reported separately. Lung function parameters were collected as exploratory assessments throughout the study.

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Kinane TB, Mayer OH, Duda PW, Lowes LP, Moody SL, Mendell JR. Long-Term Pulmonary Function in Duchenne Muscular Dystrophy: Comparison of Eteplirsen-Treated Patients to Natural History. J Neuromuscul Dis. 2017 Dec 20. [Epub ahead of print]