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One-year follow up of three Italian patients with Duchenne muscular dystrophy treated with ataluren: is earlier better?
BACKGROUND: Ataluren was approved for the treatment of nmDMD, both the efficacy and safety have been previously reported only from clinical trials but no report exists about real-life experience. PATIENT/METHODS: we describe three Italian children with nmDMD treated with ataluren for 1 year. Measure...
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| Udgivet i: | Ther Adv Neurol Disord |
|---|---|
| Main Authors: | , , , , , , , |
| Format: | Artigo |
| Sprog: | Inglês |
| Udgivet: |
SAGE Publications
2018
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| Fag: | |
| Online adgang: | https://ncbi.nlm.nih.gov/pmc/articles/PMC6236582/ https://ncbi.nlm.nih.gov/pubmed/30542376 https://ncbi.nlm.nih.govhttp://dx.doi.org/10.1177/1756286418809588 |
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