This is highly significant given that obtaining muscle biopsies from young children with severe neuromuscular diseases is challenging, performing serial biopsies to monitor progression and treatment response potentially prohibitive, and evaluating muscle biopsies can be confounded by sampling variability. 31 Furthermore, by leveraging existing investigational therapeutics, we have ascertained a significant treatment-related change in miR-133a in Mtm1 KO mice that supports its utility to streamline therapeutic development.
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microRNA-133a as an indicator of disease progression and treatment response in X-linked myotubular myopathy.
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