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PedAM

Pediatric Disease Annotations & Medicines




Disease muscular atrophy
Phenotype C0026850|muscular dystrophy
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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