Disease · MONDO_0011758

Hurler syndrome

6 sourcesdiseases-2026-09-18-0.1.0

Cite this record

ConductScience Library. Hurler syndrome. Diseases, record MONDO_0011758. Release diseases-2026-09-18-0.1.0, 18 Sept 2026. https://conductscience.org/library/diseases/MONDO_0011758

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: Hurler syndrome; Open Targets
Open Targets ID: MONDO_0011758; Open Targets
Other names: Hurler disease, MPS I H, MPS1H, MPSIH, mucopolysaccharidosis type 1H, mucopolysaccharidosis type IH; Open Targets
Disease Ontology: DOID:0111390; Disease Ontology
NCI Thesaurus: C61261; NCI Thesaurus
Orphanet: ORPHA:93473; Orphanet
Highest stage of its drugs: Phase 2, for 4 of its 7 drugs, from ClinicalTrials.gov; Open Targets

Drugs7

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 2	ClinicalTrials.gov	2	2	2	Drugs and targets
ANTILYMPHOCYTE IMMUNOGLOBULIN (HORSE)	CHEMBL2108981		Phase 2	ClinicalTrials.gov	1	1	0	Drugs and targets
BUSULFAN	CHEMBL820	Small molecule	Phase 2	ClinicalTrials.gov	1	1	1	Drugs and targets
CYCLOPHOSPHAMIDE	CHEMBL1200796	Small molecule	Phase 2	ClinicalTrials.gov	1	1	1	Drugs and targets
ALEMTUZUMAB	CHEMBL1201587	Antibody	Phase 1/2	ClinicalTrials.gov	1	1	1	Drugs and targets
FLUDARABINE	CHEMBL1568	Small molecule	Phase 1/2	ClinicalTrials.gov	1	1	1	Drugs and targets
MELPHALAN	CHEMBL852	Small molecule	Phase 1/2	ClinicalTrials.gov	1	1	1	Drugs and targets

Targets1

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
CD52	CAMPATH-1 antigen	1	CAMPATH-1 antigen inhibitor	Phase 1/2	https://conductscience.org/library/genes/1043

Source Drugs and targets

Trials4

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
NCT00176917	Stem Cell Transplantation for Hurler	Phase 2	41	2009-09-04	Mucopolysaccharidosis I, Mucopolysaccharidosis VI, Mannosidosis, Mucolipidosis Type II (I-cell Disease)	BUSULFAN, CYCLOPHOSPHAMIDE	Its record names the disease	ClinicalTrials.gov
NCT00176891	Stem Cell Transplant w/Laronidase for Hurler	Phase 2	25	2019-07-11	Mucopolysaccharidosis I, Hurler Syndrome	LARONIDASE	Its record names the disease	ClinicalTrials.gov
NCT00920972	Campath/Fludarabine/Melphalan Transplant Conditioning for Non-Malignant Diseases	Phase 1/2	220		Metabolic Disorders, Hematologic, Immune, or Bone Marrow Disorders, Hemoglobinopathies, Non-malignant Disorders	ALEMTUZUMAB, FLUDARABINE, MELPHALAN	Open Targets' reading of its record	ClinicalTrials.gov
NCT05634512	Evaluation of Intravenous Laronidase Pharmacokinetics Before and After Hematopoietic Cell Transplantation in Patients With Mucopolysaccharidosis Type IH.		13		Hematopoietic Cell Transplantation, Mucopolysaccharidosis Type I	LARONIDASE	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 trials5

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
18500373	RESULT	1	PubMed
18026148	RESULT	1	PubMed
16990602	RESULT	1	PubMed
15592491	RESULT	1	PubMed
26348869	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_0011758

GET https://conductscience.org/api/diseases/v1/MONDO_0011758/drugs

API reference For AI assistants