Disease · MONDO_0001586

mucopolysaccharidosis type 1

6 sourcesdiseases-2026-09-18-0.1.0

Cite this record

ConductScience Library. mucopolysaccharidosis type 1. Diseases, record MONDO_0001586. Release diseases-2026-09-18-0.1.0, 18 Sept 2026. https://conductscience.org/library/diseases/MONDO_0001586

On this page

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: mucopolysaccharidosis type 1; Open Targets
Open Targets ID: MONDO_0001586; Open Targets
Other names: Alpha-L-iduronidase deficiency, MPS1, MPSI, Mucopolysaccharidosis Type I, lipochondrodystrophy; Open Targets
Disease Ontology: DOID:12802; Disease Ontology
NCI Thesaurus: C85053; NCI Thesaurus
Orphanet: ORPHA:579; Orphanet
Highest stage of its drugs: Phase 3, for 1 of its 5 drugs, from ClinicalTrials.gov; Open Targets

Drugs5

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
LARONIDASE	CHEMBL1201595	Enzyme	Phase 3	ClinicalTrials.gov	5	5	5	Drugs and targets
SOMATROPIN	CHEMBL1201621	Protein	Phase 2/3	ClinicalTrials.gov	1	1	1	Drugs and targets
ADALIMUMAB	CHEMBL1201580	Antibody	Phase 1/2	ClinicalTrials.gov	2	2	2	Drugs and targets
AZATHIOPRINE	CHEMBL1542	Small molecule	Phase 1/2	ClinicalTrials.gov	1	1	1	Drugs and targets
CYCLOSPORINE	CHEMBL160	Protein	Phase 1/2	ClinicalTrials.gov	1	1	1	Drugs and targets

Targets4

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
GHR	Growth hormone receptor	1	Growth hormone receptor agonist	Phase 2/3	https://conductscience.org/library/genes/2690	Drugs and targets
PPAT	Amidophosphoribosyltransferase	1	Amidophosphoribosyltransferase inhibitor	Phase 1/2	https://conductscience.org/library/genes/5471	Drugs and targets
PPIA	Peptidyl-prolyl cis-trans isomerase A	1	Cyclophilin A modulator	Phase 1/2	https://conductscience.org/library/genes/5478	Drugs and targets
TNF	Tumor necrosis factor	1	TNF-alpha inhibitor	Phase 1/2	https://conductscience.org/library/genes/7124	Drugs and 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.

Trial	Title	Phase	Participants	Results first posted	Conditions	Drugs	Linked by	Source
NCT01173016	Administration of IV Laronidase Post Bone Marrow Transplant in Hurler	Phase 1	11	2020-03-20	Hurler Syndrome	LARONIDASE	Its record names the disease	ClinicalTrials.gov
NCT00741338	Immune Tolerance Study With Aldurazyme® (Laronidase)	Phase 1/2	7	2014-07-02	Mucopolysaccharidosis I	AZATHIOPRINE, CYCLOSPORINE, LARONIDASE	Its record names the disease	ClinicalTrials.gov
NCT00748969	Clinical Trial of Growth Hormone in MPS I, II, and VI	Phase 2/3	2	2014-11-24	Mucopolysaccharidosis I, Mucopolysaccharidosis II, Mucopolysaccharidosis VI	SOMATROPIN	Its record names the disease	ClinicalTrials.gov
NCT02437253	Effects of Adalimumab in Mucopolysaccharidosis Types I, II and VI	Phase 1/2	2	2017-09-15	Mucopolysaccharidosis Type I, Mucopolysaccharidosis Type II, Mucopolysaccharidosis Type VI	ADALIMUMAB	Its record names the disease	ClinicalTrials.gov
NCT00638547	Intrathecal Enzyme Replacement for Hurler Syndrome	Phase 1	26		Hurler Syndrome	LARONIDASE	Its record names the disease	ClinicalTrials.gov
NCT01572636	Laronidase (Aldurazyme TM) Enzyme Replacement Therapy With Hematopoietic Stem Cell Transplant for Hurler Syndrome		20		Mucopolysaccharidosis Type IH, MPS I, Hurler Syndrome	LARONIDASE	Its record names the disease	ClinicalTrials.gov
NCT03153319	Study to Evaluate the Safety and Efficacy of Adalimumab in MPS I, II, and VI	Phase 1/2	14		Mucopolysaccharidosis I, Mucopolysaccharidosis II, Mucopolysaccharidosis VI	ADALIMUMAB	Its record names the disease	ClinicalTrials.gov
NCT06406153	Efficacy and Safety of YW17 (Laronidase-CinnaGen) Compared to Aldurazyme® in MPS I Patients	Phase 3	12		Mucopolysaccharidosis Type 1	LARONIDASE	Its record names the disease	ClinicalTrials.gov

Comparisons in its trials6

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.

Comparison	Group 1, with the outcome	Group 2, with the outcome	Participants	Trial	Source
Cohort 1 vs Cohort 2: Number of Participants Who Achieved Immune Tolerance Induction	0 of 3	1 of 3	6	NCT00741338	Trial comparisons
Cohort 1 vs Cohort 2: serious adverse events	2 of 3	1 of 3	6	NCT00741338	Trial comparisons
Adalimumab vs Placebo: deaths from any cause	0 of 2	0 of 2	4	NCT02437253	Trial comparisons
Adalimumab vs Placebo: serious adverse events	0 of 2	0 of 2	4	NCT02437253	Trial comparisons
Adalimumab First, Then Placebo vs Placebo First, Then Adalimumab: Anti-ERT Antibodies	0 of 1	0 of 1	2	NCT02437253	Trial comparisons
Adalimumab First, Then Placebo vs Placebo First, Then Adalimumab: Pain Measured by the Visual Analog Scale (VAS) in the Pediatric Pain Questionnaire (PPQ)	0 of 1	1 of 1	2	NCT02437253	Trial comparisons

Papers from its trials3

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
40830667	DERIVED	1	PubMed
31575939	DERIVED	1	PubMed
28119821	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_0001586

GET https://conductscience.org/api/diseases/v1/MONDO_0001586/drugs

API reference For AI assistants