RTC13 restores dystrophin expression and improves muscle function in the mdx mouse model for Duchenne muscular dystrop (DMD)[1].
| Animal Model: | Wild-type (C57BL/10) mice at 10 to 12 weeks of age[1]. |
| Dosage: | 10 (n = 5), 30 (n = 5), 60 (n = 5) or 300 mg/kg (n = 5). |
| Administration: | Intraperitoneal Administration. |
| Result: | No changes were observed in liver and kidney weights at the time of necropsies, and Hematoxylin and Eosin (H&E) staining revealed the absence of histopathological changes. |
[1]. Refik Kayali, et al. Read-through compound 13 restores dystrophin expression and improves muscle function in the mdx mouse model for Duchenne muscular dystrop. Hum Mol Genet. 2012 Sep 15;21(18):4007-20.