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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.

Author information

1
Department of Pediatric Neurology, Catholic University, Rome, Italy.
2
Centro Clinico Nemo, Policlinico Universitario A Gemelli IRCCS, Rome, Italy.
3
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.
4
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.
5
Hospital Sant Joan de Déu Unidad de Patología Neuromuscular, Universidad de Barcelona, CIBERER, ISCIII, Barcelona, Spain.
6
Department of Pediatrics, Gothenburg University, Queen Silvia Children's Hospital, Gothenburg, Sweden.
7
Parent Project Italy APS, Rome, Italy.
8
Therapeutic Research in Neuromuscular Disorders Solutions, Pittsburgh, PA, USA.
9
Center for Genetic Medicine, Children's National Health System & the George Washington, Washington, DC, USA.
10
PTC Therapeutics Inc., South Plainfield, NJ 07080-2449, USA.
11
University of California Davis School of Medicine, Davis, CA, USA.
12
APHP Necker - Enfants Malades Hospital, Paris V Descartes University, Neuromuscular Network FILNEMUS, Paris, France.

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. ClinicalTrials.gov identifier: NCT02369731.

KEYWORDS:

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

PMID:
31997646
DOI:
10.2217/cer-2019-0171
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