Mercuri, E;
Muntoni, F;
Osorio, AN;
Tulinius, M;
Buccella, F;
Morgenroth, LP;
Gordish-Dressman, H;
... CINRG Duchenne Natural History Investigators; + view all
(2020)
Safety and effectiveness of ataluren: comparison of results from the STRIDE Registry and CINRG DMD Natural History Study.
Journal of Comparative Effectiveness Research
10.2217/cer-2019-0171.
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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.
| Type: | Article |
|---|---|
| Title: | Safety and effectiveness of ataluren: comparison of results from the STRIDE Registry and CINRG DMD Natural History Study |
| Location: | England |
| Open access status: | An open access version is available from UCL Discovery |
| DOI: | 10.2217/cer-2019-0171 |
| Publisher version: | https://doi.org/10.2217/cer-2019-0171 |
| Language: | English |
| Additional information: | This work is licensed under the Attribution-NonCommercial-NoDerivatives 4.0 Unported License. To view a copy of this license, visit http://creativecommons.org/licenses/by-nc-nd/4.0/ |
| Keywords: | STRIDE Registry, ataluren, dystrophin, effectiveness, nonsense mutation Duchenne muscular dystrophy, safety |
| UCL classification: | UCL UCL > Provost and Vice Provost Offices > School of Life and Medical Sciences UCL > Provost and Vice Provost Offices > School of Life and Medical Sciences > Faculty of Population Health Sciences > UCL GOS Institute of Child Health UCL > Provost and Vice Provost Offices > School of Life and Medical Sciences > Faculty of Population Health Sciences > UCL GOS Institute of Child Health > Developmental Neurosciences Dept |
| URI: | https://discovery.ucl.ac.uk/id/eprint/10091291 |
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