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J Comp Eff Res. 2020 Jan 30. doi: 10.2217/cer-2019-0171. [Epub ahead of print]

Safety and effectiveness of ataluren: comparison of results from the STRIDE Registry and CINRG DMD Natural History Study.

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Department of Pediatric Neurology, Catholic University, Rome, Italy.
Centro Clinico Nemo, Policlinico Universitario A Gemelli IRCCS, Rome, Italy.
Dubowitz Neuromuscular Centre & MRC Centre for Neuromuscular Diseases, University College London, Institute of Child Health & Great Ormond Street Hospital for Children Foundation Trust, 30 Guildford Street, London, WC1N 1EH, UK.
NIHR Great Ormond Street Hospital Biomedical Research Centre, Great Ormond Street Institute of Child Health, University College London, Great Ormond Street Hospital Trust, London, UK.
Hospital Sant Joan de Déu Unidad de Patología Neuromuscular, Universidad de Barcelona, CIBERER, ISCIII, Barcelona, Spain.
Department of Pediatrics, Gothenburg University, Queen Silvia Children's Hospital, Gothenburg, Sweden.
Parent Project Italy APS, Rome, Italy.
Therapeutic Research in Neuromuscular Disorders Solutions, Pittsburgh, PA, USA.
Center for Genetic Medicine, Children's National Health System & the George Washington, Washington, DC, USA.
PTC Therapeutics Inc., South Plainfield, NJ 07080-2449, USA.
University of California Davis School of Medicine, Davis, CA, USA.
APHP Necker - Enfants Malades Hospital, Paris V Descartes University, Neuromuscular Network FILNEMUS, Paris, France.


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. identifier: NCT02369731.


STRIDE Registry; ataluren; dystrophin; effectiveness; nonsense mutation Duchenne muscular dystrophy; safety

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