Disease · MONDO_0018937

mucopolysaccharidosis type 3

6 sourcesdiseases-2026-09-18-0.1.0

Cite this record

ConductScience Library. mucopolysaccharidosis type 3. Diseases, record MONDO_0018937. Release diseases-2026-09-18-0.1.0, 18 Sept 2026. https://conductscience.org/library/diseases/MONDO_0018937

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 3; Open Targets
Open Targets ID: MONDO_0018937; Open Targets
Other names: MPS3, MPSIII, Mucopoly-saccharidosis type 3, Mucopolysaccharidosis Type III, Sanfilippo disease, Sanfilippo syndrome, heparan sulphate sulfatase deficiency; Open Targets
Disease Ontology: DOID:12801; Disease Ontology
NCI Thesaurus: C61262; NCI Thesaurus
Orphanet: ORPHA:581; Orphanet
Highest stage of its drugs: Phase 2/3, for 2 of its 11 drugs, from ClinicalTrials.gov; Open Targets

Drugs11

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
AMBROXOL	CHEMBL153479	Small molecule	Phase 2/3	ClinicalTrials.gov	1	1	0	Drugs and targets
CANNABIDIOL	CHEMBL190461	Small molecule	Phase 2/3	ClinicalTrials.gov	1	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
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
MYCOPHENOLATE MOFETIL	CHEMBL1456	Small molecule	Phase 2	ClinicalTrials.gov	1	1	1	Drugs and targets
N-SULFOGLUCOSAMINE SULFOHYDROLASE RECOMBINANT	CHEMBL4297976	Enzyme	Phase 2	TTD	2	0	0	Drugs and targets
ANAKINRA	CHEMBL1201570	Protein	Phase 1/2	ClinicalTrials.gov	1	1	1	Drugs and targets
SOBI-003	CHEMBL4298107		Phase 1/2	TTD	1	0	0	Drugs and targets

1 to 10 of 11

See all 11 drugs

Targets19

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
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
CD52	CAMPATH-1 antigen	1	CAMPATH-1 antigen inhibitor	Phase 2	https://conductscience.org/library/genes/1043	Drugs and targets
CNR1	Cannabinoid receptor 1	1	Cannabinoid CB1 receptor negative allosteric modulator	Phase 2/3	https://conductscience.org/library/genes/1268	Drugs and targets
IL1R1	Interleukin-1 receptor type 1	1	Interleukin-1 receptor antagonist	Phase 1/2	https://conductscience.org/library/genes/3554	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
IMPDH2	Inosine-5'-monophosphate dehydrogenase (IMPDH)	1	Inosine-5'-monophosphate dehydrogenase (IMPDH) inhibitor	Phase 2	https://conductscience.org/library/genes/3615	Drugs and targets
POLA1	DNA polymerase (alpha/delta/epsilon)	1	DNA polymerase (alpha/delta/epsilon) inhibitor	Phase 2	https://conductscience.org/library/genes/5422	Drugs and targets
POLA2	DNA polymerase (alpha/delta/epsilon)	1	DNA polymerase (alpha/delta/epsilon) inhibitor	Phase 2	https://conductscience.org/library/genes/23649	Drugs and targets

1 to 10 of 19

See all 19 targets

Trials3

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
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
NCT06333041	Study of Cannabidiol in Sanfilippo Syndrome	Phase 2/3	35		Sanfilippo Syndrome, Mucopolysaccharidosis III	CANNABIDIOL	Its record names the disease	ClinicalTrials.gov
NCT04018755	Open-label Study of Anakinra in MPS III	Phase 1/2	24		Mucopolysaccharidosis III	ANAKINRA	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
38907160	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_0018937

GET https://conductscience.org/api/diseases/v1/MONDO_0018937/drugs

API reference For AI assistants