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Published on: May 12, 2013
Adenylosuccinic Acid: An Orphan Drug with Untapped Potential
Emma Rybalka1,2,3,4, Stephanie Kourakis1,2, Charles A Bonsett5,6
1Institute for Health and Sport (IHeS), Victoria University, Melbourne, VIC 8001, Australia.
Abstract:
Adenylosuccinic acid (ASA) is an orphan drug that was once investigated for clinical application in Duchenne muscular dystrophy (DMD). Endogenous ASA participates in purine recycling and energy homeostasis but might also be crucial for averting inflammation and other forms of cellular stress during intense energy demand and maintaining tissue biomass and glucose disposal. This article documents the known biological functions of ASA and explores its potential application for the treatment of neuromuscular and other chronic diseases.
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