Long term treatment with ataluren—the Swedish experience

INTRODUCTION: Ataluren is a relatively new treatment for male patients with Duchenne muscular dystrophy (DMD) due to a premature stop codon. Long-term longitudinal data as well as efficacy data on non-ambulant patients are still lacking. Here we present the results from a long-term follow-up study o...

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Detalles Bibliográficos
Autores principales: Michael, Eva, Sofou, Kalliopi, Wahlgren, Lisa, Kroksmark, Anna-Karin, Tulinius, Már
Formato: Online Artículo Texto
Lenguaje:English
Publicado: BioMed Central 2021
Materias:
Research
Acceso en línea:https://www.ncbi.nlm.nih.gov/pmc/articles/PMC8485550/
https://www.ncbi.nlm.nih.gov/pubmed/34592975
http://dx.doi.org/10.1186/s12891-021-04700-z

Internet

https://www.ncbi.nlm.nih.gov/pmc/articles/PMC8485550/
https://www.ncbi.nlm.nih.gov/pubmed/34592975
http://dx.doi.org/10.1186/s12891-021-04700-z

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