Enzyme replacement therapy shows promising results in X-linked myotubular myopathy
A collaborative research team including a Medical College of Wisconsin (MCW) pediatric neuropathologist successfully mitigated some of the effects of a muscular disease by using a new targeted enzyme replacement therapy strategy from 4s3 Bioscience.
The findings are published in the January edition of Human and Molecular Genetics http://hmg.oxfordjournals.org/content/early/2013/01/09/hmg.ddt003.full.pdf+html.
X-linked myotubular myopathy (XLMTM) is a severe muscle disease caused by an absence of a protein called myotubularin. There is currently no treatment for this ...







