Disease · MONDO_0018544

adrenoleukodystrophy

6 sourcesdiseases-2026-09-18-0.1.0

Cite this record

ConductScience Library. adrenoleukodystrophy. Diseases, record MONDO_0018544. Release diseases-2026-09-18-0.1.0, 18 Sept 2026. https://conductscience.org/library/diseases/MONDO_0018544

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Disease

Names and IDs as Open Targets gives them. Its IDs in MeSH, Orphanet, the Disease Ontology and the NCI Thesaurus are those Open Targets states for it.

Name: adrenoleukodystrophy; Open Targets
Open Targets ID: MONDO_0018544; Open Targets
Other names: ABCD1 deficiency, ALD, Bronze-Schilder disease, Siemerling-Creutzfeldt disease, X-ALD, X-Linked Adrenoleukodystrophy, X-linked ALD, adrenoleukodystrophy, X-linked, adrenoleukodystrophy, X-linked recessive, adrenomyeloneuropathy, adult, adrenomyeloneuropathy, adult, X-linked recessive, diffuse cerebral sclerosis of Schilder; Open Targets
Disease Ontology: DOID:10588; Disease Ontology
MeSH: D000326; MeSH
NCI Thesaurus: C61252; NCI Thesaurus
Orphanet: ORPHA:43; Orphanet
Highest stage of its drugs: Approval, for 1 of its 18 drugs, from EMA Human Drugs, at withdrawal; Open Targets

Drugs18

The drugs Open Targets' clinical reports study for the disease, the highest stage first, then the most trial reports. The stage is Open Targets', the highest any of its sources reports for the pair, and each row names the sources that report it.

Drug	ChEMBL ID	Type	Open Targets' stage	Stage reported by	Reports	Of them trials	Trials shown here	Source
ELIVALDOGENE AUTOTEMCEL	CHEMBL4594333	Gene	Approval	EMA Human Drugs, at withdrawal	2	0	0	Drugs and targets
ELIVALDOGENE TAVALENTIVEC	CHEMBL3990046	Gene	Phase 3	TTD	1	0	0	Drugs and targets
ALBUMIN HUMAN	CHEMBL1201451	Protein	Phase 2/3	ClinicalTrials.gov	1	1	1	Drugs and targets
GLYCERYL TRIERUCATE	CHEMBL4297655	Small molecule	Phase 2	ClinicalTrials.gov	2	2	1	Drugs and targets
LERIGLITAZONE	CHEMBL1267	Small molecule	Phase 2	ClinicalTrials.gov	3	1	1	Drugs and targets
ALEMTUZUMAB	CHEMBL1201587	Antibody	Phase 2	ClinicalTrials.gov	1	1	1	Drugs and targets
CLOFARABINE	CHEMBL1750	Small molecule	Phase 2	ClinicalTrials.gov	1	1	1	Drugs and targets
GLYCERYL TRIOLEATE	CHEMBL4297656	Small molecule	Phase 2	ClinicalTrials.gov	1	1	1	Drugs and targets
HYDROXYUREA	CHEMBL467	Small molecule	Phase 2	ClinicalTrials.gov	1	1	1	Drugs and targets
MELPHALAN	CHEMBL852	Small molecule	Phase 2	ClinicalTrials.gov	1	1	1	Drugs and targets

1 to 10 of 18

See all 18 drugs

Targets25

The targets ChEMBL's mechanism records give for the disease's drugs, through Open Targets, the most drugs first. A mechanism record says what a drug acts on, not that the target causes the disease.

Target	Protein	Its drugs that act on it	Mechanisms	Highest stage of those drugs	Gene in Genes	Source
PPARG	Peroxisome proliferator-activated receptor	3	Peroxisome proliferator-activated receptor agonist; Peroxisome proliferator-activated receptor gamma agonist	Phase 2	https://conductscience.org/library/genes/5468	Drugs and targets
RRM1	Ribonucleoside-diphosphate reductase RR1	2	Ribonucleoside-diphosphate reductase RR1 inhibitor	Phase 2	https://conductscience.org/library/genes/6240	Drugs and targets
RRM2	Ribonucleoside-diphosphate reductase RR1	2	Ribonucleoside-diphosphate reductase RR1 inhibitor	Phase 2	https://conductscience.org/library/genes/6241	Drugs and targets
RRM2B	Ribonucleoside-diphosphate reductase RR1	2	Ribonucleoside-diphosphate reductase RR1 inhibitor	Phase 2	https://conductscience.org/library/genes/50484	Drugs and targets
ABCD1	ABCD1	1	ABCD1 exogenous gene	Approval	https://conductscience.org/library/genes/215	Drugs and targets
CD52	CAMPATH-1 antigen	1	CAMPATH-1 antigen inhibitor	Phase 2	https://conductscience.org/library/genes/1043	Drugs and targets
CRBN	CRL4(CRBN) E3 ubiquitin ligase	1	CRL4(CRBN) E3 ubiquitin ligase inhibitor	Unknown	https://conductscience.org/library/genes/51185	Drugs and targets
CUL4A	CRL4(CRBN) E3 ubiquitin ligase	1	CRL4(CRBN) E3 ubiquitin ligase inhibitor	Unknown	https://conductscience.org/library/genes/8451	Drugs and targets
DDB1	CRL4(CRBN) E3 ubiquitin ligase	1	CRL4(CRBN) E3 ubiquitin ligase inhibitor	Unknown	https://conductscience.org/library/genes/1642	Drugs and targets
IMPDH1	Inosine-5'-monophosphate dehydrogenase (IMPDH)	1	Inosine-5'-monophosphate dehydrogenase (IMPDH) inhibitor	Phase 2	https://conductscience.org/library/genes/3614	Drugs and targets

1 to 10 of 25

See all 25 targets

Trials8

Trials an Open Targets clinical report links to the disease, posted results first, then the largest. A trial is shown when the release's checks confirm its link to the disease and Drugs and targets shows its link to one of the disease's drugs. 1 more is not shown, since the checks do not confirm it.

Trial	Title	Phase	Participants	Results first posted	Conditions	Drugs	Linked by	Source
NCT00004418	Effect of Glycerol Trierucate on Clinical Course of Adrenoleukodystrophy	Phase 2	126	2020-10-23	Adrenoleukodystrophy	GLYCERYL TRIERUCATE, GLYCERYL TRIOLEATE	Its record names the disease	ClinicalTrials.gov
NCT00383448	HSCT for High Risk Inherited Inborn Errors	Phase 2	38	2019-07-11	Adrenoleukodystrophy, Metachromatic Leukodystrophy, Globoid Cell Leukodystrophy, Tay Sachs Disease, Sandhoffs Disease, Wolman Disease, I-Cell Disease, Sanfilippo Syndrome, GM1 Gangliosidosis	ALEMTUZUMAB, CLOFARABINE, HYDROXYUREA, MELPHALAN, MYCOPHENOLATE MOFETIL	Its record names the disease	ClinicalTrials.gov
NCT00004450	Randomized Study of Beta Interferon and Thalidomide in Patients With Adrenoleukodystrophy	Not applicable	60		Adrenoleukodystrophy	INTERFERON BETA, THALIDOMIDE	Its record names the disease	ClinicalTrials.gov
NCT03864523	Effect of Pioglitazone Administered to Patients With Adrenomyeloneuropathy	Phase 2	18		Adrenomyeloneuropathy, X-linked Adrenoleukodystrophy	PIOGLITAZONE	Its record names the disease	ClinicalTrials.gov
NCT04528706	A Clinical Study in Male Pediatric Patients With Cerebral X-linked Adrenoleukodystrophy (Cald) to Assess the Effects of MIN-102 Treatment on Disease Progression Prior to Human Stem Cell Transplant (HSCT)	Phase 2	13		Cerebral Adrenoleukodystrophy	LERIGLITAZONE	Its record names the disease	ClinicalTrials.gov
NCT01165060	The Effect of Bezafibrate on the Level of Very Long Chain Fatty Acids (VLCFA) in X-linked Adrenoleukodystrophy (X-ALD)	Not applicable	10		X-linked Adrenoleukodystrophy, Adrenomyeloneuropathy	BEZAFIBRATE	Its record names the disease	ClinicalTrials.gov
NCT04303416	Plasma Exchange With Albumin in AMN Patients	Phase 2/3	5		Adrenomyeloneuropathy, Adrenoleukodystrophy	ALBUMIN HUMAN	Its record names the disease	ClinicalTrials.gov
NCT01787578	Safety and Pharmacodynamic Study of Sobetirome in X-Linked Adrenoleukodystrophy (X-ALD)	Phase 1	0		X-Linked Adrenoleukodystrophy, Adrenomyeloneuropathy	SOBETIROME	Its record names the disease	ClinicalTrials.gov

Comparisons in its trials0

Comparisons Trial comparisons calculated in the trials shown here, most participants first. A comparison in one of these trials does not always compare a treatment for the disease, so read the two groups.

None in this release.

Papers from its trials2

The papers the trials shown here list on ClinicalTrials.gov as their results (RESULT) or as drawn from them (DERIVED), results first, newest first.

PMID	Listed as	Trials listing it	Source
22911730	DERIVED	1	PubMed
21586746	DERIVED	1	PubMed

Data dates

The date of each source this release read.

Open Targets: 2026-09-18; Open Targets
Drug links and their checks, from Drugs and targets: 2026-09-18; Drugs and targets
ClinicalTrials.gov, from Trials: 2026-11-12; Trials
Comparisons, from Trial comparisons: 2026-03-23; Trial comparisons
PubMed, for the checks: 2026-11-06; PubMed
MeSH, for the checks: 2026-11-10; MeSH

Sources 6

ClinicalTrials.gov's posted results, through CTTI's AACTFreely available. Acknowledgement requested
ChEMBL, through Open TargetsCC BY-SA 3.0
ClinicalTrials.govNo license stated
NLM's MeSHNLM's terms
Open TargetsCC0 1.0
PubMedNLM's terms

Each source's terms

ClinicalTrials.gov's posted results, through CTTI's AACTClinicalTrials.gov data via Aggregate Analysis of ClinicalTrials.gov (AACT), CTTI. CTTI makes its materials freely available and requests acknowledgement. https://aact.ctti-clinicaltrials.org/documentation/299
ChEMBL, through Open TargetsCC BY-SA 3.0, for the parts from ChEMBL. Read on 8 Nov 2026 at https://platform-docs.opentargets.org/licence
ClinicalTrials.govnone stated
NLM's MeSHNLM's
Open TargetsCC0 1.0. Open Targets asks that users cite its latest paper: Buniello et al. 2025, Open Targets Platform: facilitating therapeutic hypotheses building in drug discovery, Nucleic Acids Research 53(D1):D1467, PMID 39657122. Read on 8 Nov 2026 at https://platform-docs.opentargets.org/licence
PubMedNLM's terms

ClinicalTrials.gov's data carry an international copyright outside the United States: https://clinicaltrials.gov/about-site/terms-conditions.

For software

Read this disease from software.

The record and each of its lists are JSON, twenty rows a page. No key.

GET https://conductscience.org/api/diseases/v1/MONDO_0018544

GET https://conductscience.org/api/diseases/v1/MONDO_0018544/targets?page=2

API reference For AI assistants