Population
Patients with neuromuscular diseases and healthy controls, as well as a congenital myotubular myopathy mouse…
Comparison
In mice: Intramuscular injection of AAV-PropD76A… vs In mice: Intramuscular injection of sterile PBS…
Design
Preclinical, Animal were not randomized for allocation in the experimental…
Authors
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Questions myostatin inhibitor use in dystrophies; leaves open alternative targets for muscle wasting.
The downregulation of the myostatin pathway in severe neuromuscular diseases may explain the limited clinical efficacy of anti-myostatin therapies, suggesting that correcting the primary defect may be required for these therapies to be effective.
Mariot et al. (2017) studied this question.
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