Adenylosuccinic Acid: An Orphan Drug with Untapped Potential.

ADSSL1 myopathy Duchenne muscular dystrophy Nrf2 activation adenylosuccinate adenylosuccinic acid immunometabolism metabolic disease purine metabolism skeletal muscle succinyl-AMP

Journal

Pharmaceuticals (Basel, Switzerland)
ISSN: 1424-8247
Titre abrégé: Pharmaceuticals (Basel)
Pays: Switzerland
ID NLM: 101238453

Informations de publication

Date de publication:
31 May 2023
Historique:
received: 09 05 2023
revised: 24 05 2023
accepted: 24 05 2023
medline: 28 6 2023
pubmed: 28 6 2023
entrez: 28 6 2023
Statut: epublish

Résumé

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.

Identifiants

pubmed: 37375769
pii: ph16060822
doi: 10.3390/ph16060822
pmc: PMC10304260
pii:
doi:

Types de publication

Journal Article

Langues

eng

Subventions

Organisme : NICHD NIH HHS
ID : P50 HD105351
Pays : United States

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Auteurs

Emma Rybalka (E)

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.
Department of Medicine-Western Health, Melbourne Medical School, The University of Melbourne, St Albans, VIC 3021, Australia.
Division of Neuropaediatrics and Developmental Medicine, University Children's Hospital of Basel (UKBB), 4056 Basel, Switzerland.

Stephanie Kourakis (S)

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.

Charles A Bonsett (CA)

Dystrophy Concepts Incorporated, Indianapolis, IN 46226, USA.
School of Medicine, Indiana University, Indianapolis, IN 46202, USA.

Behzad Moghadaszadeh (B)

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

Alan H Beggs (AH)

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

Cara A Timpani (CA)

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

Classifications MeSH