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PedAM

Pediatric Disease Annotations & Medicines




Disease muscular atrophy
Phenotype C0013264|duchenne muscular dystrophy
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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.

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