Safety, Tolerability, and Pharmacokinetics of SMT C1100, a 2-Arylbenzoxazole Utrophin Modulator, following Single- and Multiple-Dose Administration to Pediatric Patients with Duchenne Muscular Dystrophy

PURPOSE: SMT C1100 is a utrophin modulator being evaluated as a treatment for Duchenne muscular dystrophy (DMD). This study, the first in pediatric DMD patients, reports the safety, tolerability and PK parameters of single and multiple doses of SMT C1100, as well as analyze potential biomarkers of m...

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Vydáno v:PLoS One
Hlavní autoři: Ricotti, Valeria, Spinty, Stefan, Roper, Helen, Hughes, Imelda, Tejura, Bina, Robinson, Neil, Layton, Gary, Davies, Kay, Muntoni, Francesco, Tinsley, Jonathon
Médium: Artigo
Jazyk:Inglês
Vydáno: Public Library of Science 2016
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Research Article
On-line přístup:https://ncbi.nlm.nih.gov/pmc/articles/PMC4824384/
https://ncbi.nlm.nih.gov/pubmed/27055247
https://ncbi.nlm.nih.govhttp://dx.doi.org/10.1371/journal.pone.0152840
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