Evidence mapPaperPMID 37375769Full record

ArticlePharmaceuticals (Basel, Switzerland)2023

Adenylosuccinic Acid: An Orphan Drug with Untapped Potential.

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

Registry-linked trialOpen access · goldAbstract read
In one paragraph

Article in Pharmaceuticals (Basel, Switzerland), 2023. The graph could read no effect estimate from its abstract, so it casts no vote on the map. It is linked to trial NCT07412821 (A Phase 1b, Open Label, Single and Multiple Ascending Dose-escalation Study to Assess the Safety, Tolerability, Pharmacokinetics and Preliminary Efficacy of Subcutaneous Adenylosuccinic Acid), which is not on this map. Cited by 6 papers.

0numbers the graph read from it
0cells of the map it votes in
6citing papers in PubMed
3.5field-weighted citation impact, top 7% of its field
1 · What the graph read from it

What it found

Each row is one number read from the abstract, on the scale the paper reported it, with its interval. Left of the dashed line favours the treatment, right favours the comparator. Under each row is the sentence it came from. New to these charts? A ten-minute tutorial.

The abstract states no effect estimate the extractor could read, or names no intervention and outcome on the map, so this paper lights no cell and moves no belief. It is still indexed, cited and linked below.

2 · The registry

The trial behind it

Trials whose registry record cites this paper, or whose number appears in the abstract. A trial that started after this paper was published is citing it as background, not reporting it.

NCT07412821 phase1enrolling by invitationnot on this mapstarted 2026, after this paper: background citation

A Phase 1b, Open Label, Single and Multiple Ascending Dose-escalation Study to Assess the Safety, Tolerability, Pharmacokinetics and Preliminary Efficacy of Subcutaneous Adenylosuccinic Acid (ASA) in Two Siblings With Adenylosuccinate Synthase 1 (ADSS1) Deficient Myopathy.

TypeinterventionalSponsorCure ADSSL1Ran2026 to 2027Enrolled2ConditionsAdenylosuccinate Synthase 1 Deficient MyopathyArmsadenylosuccinic acid
3 · Its place in the literature

Who cites it

6 citing papers in PubMed, 12 citations in OpenAlex.

  1. Article
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  3. Review
  4. Article
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4 · The record

Corrections and comments

PubMed lists nothing against this paper. Absence here is not a guarantee, only a check that was made.

5 · Who and what money

Authors and funding

6 authors at 4 institutions in 3 countries.

Emma RybalkaInstitute for Health and Sport (IHeS), Victoria University, Melbourne, VIC 8001, Australia.ORCID 0000-0002-4854-0036
Stephanie KourakisInstitute for Health and Sport (IHeS), Victoria University, Melbourne, VIC 8001, Australia.ORCID 0000-0001-5683-9989
Charles A BonsettDystrophy Concepts Incorporated, Indianapolis, IN 46226, USA.
Behzad MoghadaszadehThe Manton Center for Orphan Disease Research, Division of Genetics and Genomics, Boston Children's Hospital, Harvard Medical School, Boston, MA 02115, USA.ORCID 0000-0001-9787-0106
Alan H BeggsThe Manton Center for Orphan Disease Research, Division of Genetics and Genomics, Boston Children's Hospital, Harvard Medical School, Boston, MA 02115, USA.ORCID 0000-0001-8818-0568
Cara A TimpaniInstitute for Health and Sport (IHeS), Victoria University, Melbourne, VIC 8001, Australia.ORCID 0000-0003-4567-4319
Boston Children's Hospital · USThe University of Melbourne · AUAustralian Institute for Musculoskeletal Science · AUIndiana University – Purdue University Indianapolis · US

Funding

Cellular Imaging Core (CIC)P50HD105351 · BOSTON CHILDREN'S HOSPITAL · 2025 to 2025
$1.4M
NICHD NIH HHS P50 HD105351
6 · The paper itself

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.

Indexed as

adenylosuccinateadenylosuccinic acidADSSL1 myopathyDuchenne muscular dystrophyimmunometabolismmetabolic diseaseNrf2 activationpurine metabolismskeletal musclesuccinyl-AMP

Identifiers

PMID37375769
PMCPMC10304260
OpenAlexW4378982526

What Socratic holds

Textmetadata
LicenceCC BY
Read underepoch 390

Registered trials

Read under generation 80e0d062 · epoch 390. Bibliography from PubMed, PubMed Central and OpenAlex; grants from NIH RePORTER; trial links from ClinicalTrials.gov; estimates, votes and beliefs from the Socratic graph.