• Ataluren, a medicine that targets DMD gene stop codon-type abnormalities, approved in Europe in Duchenne muscular dystrophy, is the subject of new results with respect to walking, upper limb mobility and breathing, based on real-life data from the STRIDE registry and a retrospective Swedish study
• The results show that ataluren delays the loss of ability to walk by a mean period of 2 years:
- more than 80% of 12-year-old children from the STRIDE registry (cohort of 241 treated children) maintained their ability to walk after 5 years of treatment, versus only 52% in the external control cohort of untreated patients, from the CINRG natural history study.
- The mean age for loss of ability to walk was 13.2 years in the Swedish study (11 patients), i.e. 2 years later than untreated boys (CINRG external cohort).
• Upper limb, arm and hand, function is maintained, long after loss of ability to walk, according to the Swedish study
STRIDE Data Show Translarna™ Delays Loss of Ambulation by More Than Five Years in Boys with Nonsense Mutation Duchenne Muscular Dystrophy, Press release, PTC Therapeutics, 2021/09/20.
