Myostatin expression levels in neuromuscular diseases participates in anti-myostatin clinical failure


Myostatin being a negative regulator of muscle mass, its down-regulation has been seen as a promising tool to counterbalance the muscle wasting observed in neuromuscular patients. However, so far, the clinical trials have been very disappointing and clinical endpoints have been barely reached. Different possibilities could explain this absence of functional improvement and in our study, we have analyzed the expression levels of different players involved in the myostatin pathway. We have observed that very low levels of myostatin pathway were observed at both protein and mRNA levels in the sera or muscle biopsies of neuromuscular patients, indicating that myostatin pathway is strongly down regulated in the most atrophying diseases such as Duchenne muscular disease.


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