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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Detalles Bibliográficos
Autores principales: Ricotti, Valeria, Spinty, Stefan, Roper, Helen, Hughes, Imelda, Tejura, Bina, Robinson, Neil, Layton, Gary, Davies, Kay, Muntoni, Francesco, Tinsley, Jonathon
Formato: Online Artículo Texto
Lenguaje:English
Publicado: Public Library of Science 2016
Materias:
Research Article
Acceso en línea:https://www.ncbi.nlm.nih.gov/pmc/articles/PMC4824384/
https://www.ncbi.nlm.nih.gov/pubmed/27055247
http://dx.doi.org/10.1371/journal.pone.0152840

Internet

https://www.ncbi.nlm.nih.gov/pmc/articles/PMC4824384/
https://www.ncbi.nlm.nih.gov/pubmed/27055247
http://dx.doi.org/10.1371/journal.pone.0152840

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