The STRIDE study aims to investigate the long-term real-life data of people with Duchenne muscular dystrophy with a nonsense genetic abnormality on the DMD gene and treated with ataluren (Translarna™). The latest five-year follow-up results of 307 patients aged 5 years, compared to a control external cohort (DNHS CINRG Natural History Study) were published in April 2023 :
- Ataluren significantly delayed the loss of walking by an average of four years.
- It also reduced the significant decline in forced vital capacity to <60% by 1.8 years and to <50% by 2.3 years.
The STRIDE study of ataluren, which currently benefits from conditional marketing authorisation, is due to end in 2025. New results should provide a clearer picture of whether the marketing authorisation will be maintained.
