Safety and effectiveness of ataluren in patients with nonsense mutation DMD in the STRIDE Registry compared with the CINRG Duchenne Natural History Study (2015-2022): 2022 interim analysis.
Eugenio MercuriAndrés Nascimento OsorioFrancesco MuntoniFilippo BuccellaIsabelle DesguerreJanbernd KirschnerMár TuliniusMaria Bernadete Dutra de ResendeLauren P MorgenrothHeather Gordish-DressmanShelley JohnsonAllan KristensenChristian WernerPanayiota TrifillisErik K HenricsonCraig M McDonaldnull nullPublished in: Journal of neurology (2023)
Long-term, real-world treatment with ataluren plus SoC delays several disease progression milestones in individuals with nmDMD. NCT02369731; registration date: February 24, 2015.