PedAM
Pediatric Disease Annotations & Medicines
| Disease | muscular dystrophy |
| Phenotype | C0026846|muscular atrophy |
| Sentences | 2 |
| PubMedID- 26280937 | For duchenne muscular dystrophy, spinal muscular atrophy and myotonic dystrophy trials are ongoing to assess safety and efficacy, while in parallel preclinical studies are being conducted to identify ways to improve efficiency and delivery. |
| PubMedID- 25914643 | In a mouse model of spinal muscular atrophy with a muscular dystrophy appearance of the skeletal muscle (hsa-cre, smnf7/f7 mice) receiving intravenous injection of a small number of afs cells were able to survive with drastic improvement of their muscle force (piccoli et al., 2012). |
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