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Scientific Articles

Safety and Effectiveness of Ataluren: Comparison of Results From the STRIDE Registry and CINRG DMD Natural History Study

key information

source: Journal of Comparative Effectiveness Research

year: 2020

authors: Eugenio Mercuri, Francesco Muntoni, Andrés Nascimento Osorio, Már Tulinius, Filippo Buccella, Lauren P Morgenroth, Heather Gordish-Dressman, Joel Jiang, Panayiota Trifillis, Jin Zhu, Allan Kristensen, Claudio L Santos, Erik K Henricson, Craig M McDonald, Isabelle Desguerre

summary/abstract:

Aim: 

Strategic Targeting of Registries and International Database of Excellence (STRIDE) is an ongoing, multicenter registry providing real-world evidence regarding ataluren use in patients with nonsense mutation Duchenne muscular dystrophy (nmDMD). We 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), DMD genotype–phenotype/–ataluren benefit correlations and ataluren safety.

Patients & methods:

Propensity score matching was performed to identify STRIDE and CINRG DNHS patients who were comparable in established disease progression predictors (registry cut-off date, 9 July 2018). 

Results & conclusion:

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 (p ≤ 0.05). There were no DMD genotype–phenotype/ataluren benefit correlations. Ataluren was well tolerated. These results indicate that ataluren + SoC delays functional milestones of DMD progression in patients with nmDMD in routine clinical practice.

organization: Catholic University, Rome, Italy; Policlinico Universitario A Gemelli IRCCS, Italy; University College London, UK; Universidad de Barcelona, Spain; Queen Silvia Children's Hospital, Sweden; Parent Project, Italy; Therapeutic Research in Neuromuscular Disorders Solutions, USA; Children's National Health System & the George Washington, USA; PTC Therapeutics Inc., USA; University of California Davis School of Medicine, USA; Paris V Descartes University, France

DOI: 10.2217/cer-2019-0171

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