Adenylosuccinic acid: an orphan drug with untapped potential

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Rybalka, Emma ORCID: 0000-0002-4854-0036, Kourakis, Stephanie ORCID: 0000-0001-5683-9989, Bonsett, Charles A, Moghadaszadeh, Behzad ORCID: 0000-0001-9787-0106, Beggs, Alan H ORCID: 0000-0001-8818-0568 and Timpani, Cara ORCID: 0000-0003-4567-4319 (2023) Adenylosuccinic acid: an orphan drug with untapped potential. Pharmaceuticals, 16 (6). ISSN 1424-8247


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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Item type Article
DOI 10.3390/ph16060822
Official URL
Subjects Current > FOR (2020) Classification > 3209 Neurosciences
Current > Division/Research > Institute for Health and Sport
Keywords adenylosuccinic acid, ASA, orphan drug, Duchenne muscular dystrophy, DMD, neuromuscular disease
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