Journal of Neurology · 16 authors, 15 centres
This summary was generated by AI from a single paper. It has not been reviewed by a clinician and is not clinical advice. Verify against the source before acting on it.
This interim analysis of the ongoing STRIDE Registry compared real-world ataluren plus standard of care with standard of care alone from the CINRG DNHS in patients with nonsense mutation Duchenne muscular dystrophy. Ataluren plus standard of care significantly delayed loss of ambulation by 4 years and delays to clinically significant declines in forced vital capacity compared with standard of care alone.
The STRIDE Registry is an ongoing, international, multicenter, post-approval registry of real-world ataluren use in patients with nonsense mutation Duchenne muscular dystrophy (nmDMD). This 2022 interim analysis (data cut-off January 31, 2022) enrolled 307 patients from 14 countries. Patients aged ≥2 to <5 years were included per protocol amendment, though the primary focus was on patients aged 5 years or older (n=268). Propensity score matching was used to compare STRIDE patients receiving ataluren plus standard of care with CINRG DNHS patients receiving standard of care alone. Kaplan–Meier analyses showed that ataluren plus standard of care significantly delayed the median age at loss of ambulation by 4 years (17.0 vs 13.0 years; p<0.0001), delayed decline to %-predicted forced vital capacity <60% by 1.8 years (p=0.0021), and delayed decline to <50% by 2.3 years (p=0.0207). Ataluren had a favorable safety profile; most treatment-emergent adverse events were mild or moderate and unrelated to ataluren.
