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eRAM

encyclopedia of Rare Disease Annotation for Precision Medicine




Disease muscular atrophy
Comorbidity 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.
PubMedID- 22192627 In duchenne muscular dystrophy, the absence of dystrophin causes progressive muscle wasting and premature death.

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