腺苷酸琥珀酸：一种潜力未被开发的孤儿药。

Adenylosuccinic Acid: An Orphan Drug with Untapped Potential.

作者信息

Rybalka Emma, Kourakis Stephanie, Bonsett Charles A, Moghadaszadeh Behzad, Beggs Alan H, Timpani Cara A

机构信息

Institute for Health and Sport (IHeS), Victoria University, Melbourne, VIC 8001, Australia.

Inherited and Acquired Myopathy Program, Australian Institute for Musculoskeletal Science (AIMSS), St Albans, VIC 3021, Australia.

出版信息

Pharmaceuticals (Basel). 2023 May 31;16(6):822. doi: 10.3390/ph16060822.

DOI:10.3390/ph16060822

PMID:37375769

原文链接:https://pmc.ncbi.nlm.nih.gov/articles/PMC10304260/

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.

摘要

腺苷酸琥珀酸（ASA）是一种曾被研究用于杜氏肌营养不良症（DMD）临床应用的孤儿药。内源性ASA参与嘌呤循环和能量稳态，但在能量需求旺盛时，它对于避免炎症和其他形式的细胞应激，以及维持组织生物量和葡萄糖代谢也可能至关重要。本文记录了ASA已知的生物学功能，并探讨了其在治疗神经肌肉疾病和其他慢性疾病方面的潜在应用。