Long term treatment with ataluren – the Swedish experience

A study by Michael et al. (2021) explores the long-term effects of ataluren on DMD patients’ upper motor and respiratory function. Long-term cumulative treatment outcomes over a median period of 6.3 years on ataluren treatment indicated a delay in the loss of ambulation, a slower decline in forced vital capacity (FVC) and upper limb motor function even after loss of ambulation. The Vitalograph Alpha was used to measure forced vital capacity in this study. Read the full report. Learn how our custom solutions and personalized approach meet a wide range of respiratory clinical study needs. We are flexible and work with you to develop customized solutions for the unique nature of trials in rare and orphan diseases.