PTC Therapeutics announces new data supporting benefit of ataluren
PTC Therapeutics announced new data supporting the potential benefit of ataluren in preserving lung function in non-ambulatory nonsense mutation Duchenne muscular dystrophy patients. The results, which are being presented as part of a company-sponsored symposium, are based on PTC's analyses of lung function data from one of PTC's ongoing open-label extension studies versus natural history data from a comparable non-ambulatory cohort. The analysis of ataluren's impact on preservation of lung function is based on FVC, a marker of respiratory insufficiency which progresses with age. The results presented are from a preliminary assessment of pulmonary function in non-ambulatory patients in Study 019 (n = 53). The data were compared with an external control group of patients not receiving ataluren in a long-term DMD natural history study performed by the Cooperative International Neuromuscular Research Group (n = 114). The CINRG cohort was matched using the following criteria: non-ambulatory (requiring wheelchair use), less than or equal to 25 years old, with greater than or equal to 24 months of corticosteroid use. To reflect the standard of care available during Study 019, only CINRG data from 2012 onward were included.