Adjunct therapy for Pompe disease

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Ashe K.M. et al.  Inhibition of glycogen biosynthesis via mTORC1 suppression as an adjunct therapy for Pompe disease. Molecular Genetics and Metabolism Volume 100, Issue 4, August 2010, Pages 309-315

This study reported that co-administration of the mTORC1 inhibitor rapamycin with Recombinant human GAA (rhGAA) in a GAA knockout mouse reduced muscle glycogen content more than rhGAA or rapamycin alone.

posted by Yannis Trakadis

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