A new study published in the Journal of Comparative Effectiveness Research examined the effectiveness of ataluren + standard of care (SoC) in the registry, versus SoC alone, in the Cooperative International Neuromuscular Research Group (CINRG) Duchenne Natural History Study (DNHS), Duchenne muscular dystrophy (nmDMD) genotype-phenotype/-ataluren benefit correlations and ataluren safety.

This study involved a propensity score matching, to identify STRIDE and CINRG DNHS patients who were comparable in established disease progression predictors.

Kaplan-Meier analyses demonstrated that ataluren + SoC significantly delayed age at loss of ambulation and age at worsening performance in timed function tests versus SoC alone. On the other hand, there were no DMD genotype-phenotype/ataluren benefit correlations. Besides, ataluren was well tolerated. Therefore, it was stated that ataluren + SoC delays functional milestones of DMD progression in patients with nmDMD in routine clinical practice.

Source: Journal of Comparative Effectiveness Research. 2020 Jan 30. doi: 10.2217/cer-2019-0171.