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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Detalhes bibliográficos
Publicado no:PLoS One
Main Authors: Ricotti, Valeria, Spinty, Stefan, Roper, Helen, Hughes, Imelda, Tejura, Bina, Robinson, Neil, Layton, Gary, Davies, Kay, Muntoni, Francesco, Tinsley, Jonathon
Formato: Artigo
Idioma:Inglês
Publicado em: Public Library of Science 2016
Assuntos:
Research Article
Acesso em linha: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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