References
- Mercuri E Muntoni F Osorio AN Tulinius M Buccella F Morgenroth LP STRIDE; CINRG Duchenne Natural History Investigators Safety and effectiveness of ataluren: comparison of results from the STRIDE Registry and CINRG DMD Natural History Study J Comp Eff Res 2020 Apr 9 5 341 60 https://doi.org/10.2217/cer-2019-0171 31997646
- Namgoong JH Bertoni C Clinical potential of ataluren in the treatment of Duchenne muscular dystrophy Degener Neurol Neuromuscul Dis 2016 May 6 37 48 https://doi.org/10.2147/DNND.S71808 30050367
- Muntoni F Desguerre I Guglieri M Osorio AN Kirschner J Tulinius M Ataluren use in patients with nonsense mutation Duchenne muscular dystrophy: patient demographics and characteristics from the STRIDE Registry J Comp Eff Res 2019 Oct 8 14 1187 200 https://doi.org/10.2217/cer-2019-0086 31414621
- Ciafaloni E Kumar A Liu K Pandya S Westfield C Fox DJ Age at onset of first signs or symptoms predicts age at loss of ambulation Duchenne and Becker Muscular Dystrophy: data from the MD STARnet J Pediatr Rehabil Med 2016 9 1 5 11 https://doi.org/10.3233/PRM-160361 26966795
DOI: https://doi.org/10.15844/pedneurbriefs-34-12 | Journal eISSN: 2166-6482
Language: English
Page range: 12 - 12
Submitted on: Mar 6, 2020
Accepted on: Nov 29, 2020
Published on: Dec 4, 2020
Published by: Pediatric Neurology Briefs Publishers
In partnership with: Paradigm Publishing Services
© 2020 Sameh S. Morkous, published by Pediatric Neurology Briefs Publishers
This work is licensed under the Creative Commons Attribution 4.0 License.