Adenylosuccinic Acid: An Orphan Drug with Untapped Potential-Reference-Cited by-同舟云学术

Adenylosuccinic Acid: An Orphan Drug with Untapped Potential

Published:2023-05-31 Issue:6 Volume:16 Page:822
ISSN:1424-8247
Container-title:Pharmaceuticals
language:en
Short-container-title:Pharmaceuticals

Author:

Rybalka Emma¹²³⁴^ORCID,Kourakis Stephanie¹²^ORCID,Bonsett Charles A.⁵⁶,Moghadaszadeh Behzad⁷^ORCID,Beggs Alan H.⁷^ORCID,Timpani Cara A.¹²³^ORCID

Affiliation:

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

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

3. Department of Medicine—Western Health, Melbourne Medical School, The University of Melbourne, St Albans, VIC 3021, Australia

4. Division of Neuropaediatrics and Developmental Medicine, University Children’s Hospital of Basel (UKBB), 4056 Basel, Switzerland

5. Dystrophy Concepts Incorporated, Indianapolis, IN 46226, USA

6. School of Medicine, Indiana University, Indianapolis, IN 46202, USA

7. The Manton Center for Orphan Disease Research, Division of Genetics and Genomics, Boston Children’s Hospital, Harvard Medical School, Boston, MA 02115, USA

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.

Funder

Estate of Charles A. Bonsett

Publisher

MDPI AG

Subject

Drug Discovery,Pharmaceutical Science,Molecular Medicine

Link

https://www.mdpi.com/1424-8247/16/6/822/pdf

Reference91 articles.

1. The dystrophin connection—ATP?;Bonsett;Med. Hypotheses,1992

2. Duchenne’s muscular dystrophy: A tissue culture perspective;Bonsett;Indiana Med.,1984

3. ‘Oil globules’ in Duchenne muscular dystrophy—history, demonstration, and metabolic significance;Bonsett;Med. Hypotheses,1994

4. Duchenne muscular dystrophy. A rational approach to disease comprehension and therapy;Bonsett;Indiana Med.,1986

5. Dystrophin: The protein product of the Duchenne muscular dystrophy locus;Hoffman;Cell,1987

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