McDonald, CM;
Muntoni, F;
Penematsa, V;
Jiang, J;
Kristensen, A;
Bibbiani, F;
Goodwin, E;
... Guglieri, M; + view all
(2021)
Ataluren delays loss of ambulation and respiratory decline in nonsense mutation Duchenne muscular dystrophy patients.
Journal of Comparative Effectiveness Research
10.2217/cer-2021-0196.
(In press).
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Abstract
Aim: We investigated the effect of ataluren plus standard of care (SoC) on age at loss of ambulation (LoA) and respiratory decline in patients with nonsense mutation Duchenne muscular dystrophy (nmDMD) versus patients with DMD on SoC alone. / Patients & methods: Study 019 was a long-term Phase III study of ataluren safety in nmDMD patients with a history of ataluren exposure. Propensity score matching identified Study 019 and CINRG DNHS patients similar in disease progression predictors. / Results & conclusion: Ataluren plus SoC was associated with a 2.2-year delay in age at LoA (p = 0.0006), and a 3.0-year delay in decline of predicted forced vital capacity to <60% in nonambulatory patients (p = 0.0004), versus SoC. Ataluren plus SoC delays disease progression and benefits ambulatory and nonambulatory patients with nmDMD. / ClinicalTrials.gov: NCT01557400.
| Type: | Article |
|---|---|
| Title: | Ataluren delays loss of ambulation and respiratory decline in nonsense mutation Duchenne muscular dystrophy patients |
| Open access status: | An open access version is available from UCL Discovery |
| DOI: | 10.2217/cer-2021-0196 |
| Publisher version: | https://doi.org/10.2217/cer-2021-0196 |
| Language: | English |
| Additional information: | Copyright © 2021 McDonald et al. 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: | ataluren, dystrophin, efficacy, loss of ambulation, nonsense mutation Duchenne muscular dystrophy, respiratory function, Study 019 |
| 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/10139096 |
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