Disease · MONDO_0009655
mucopolysaccharidosis type 3A
6 sourcesdiseases-2026-09-18-0.1.0
Cite this record
ConductScience Library. mucopolysaccharidosis type 3A. Diseases, record MONDO_0009655. Release diseases-2026-09-18-0.1.0, 18 Sept 2026. https://conductscience.org/library/diseases/MONDO_0009655
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.
- Other names
- MPS III A, MPS3A, MPSIIIA, Sanfilippo A, Sanfilippo syndrome a, Sanfilippo syndrome type A, heparan sulfamidase deficiency, mucopolysaccharidosis type IIIA
- Open Targets
Drugs1
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 |
|---|---|---|---|---|---|---|---|
| REBISUFLIGENE ETISPARVOVEC | CHEMBL6068466 | Gene | Phase 3 | ClinicalTrials.gov | 3 | 3 | 3 |
Source Drugs and targets
Targets0
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.
None in this release.
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.
| Trial | Title | Phase | Participants | Results first posted | Conditions | Drugs | Linked by | Source |
|---|---|---|---|---|---|---|---|---|
| NCT04088734 | Gene Transfer Study of ABO-102 in Patients With Middle and Advanced Phases of MPS IIIA Disease | Phase 1/2 | 5 | 2023-07-25 | MPS IIIA, Sanfilippo Syndrome, Sanfilippo A, Mucopolysaccharidosis III | REBISUFLIGENE ETISPARVOVEC | Its record names the disease | ClinicalTrials.gov |
| NCT04360265 | Follow-up Study of AAV-Mediated Gene Transfer (UX111; Previously Known as ABO-102) for MPS Type IIIA | Phase 3 | 41 | Mucopolysaccharidosis IIIA, MPS IIIA, Sanfilippo Syndrome, Sanfilippo A | REBISUFLIGENE ETISPARVOVEC | Its record names the disease | ClinicalTrials.gov | |
| NCT02716246 | Phase I/II/III Gene Transfer Clinical Trial of scAAV9.U1a.hSGSH | Phase 2/3 | 36 | MPS IIIA, Sanfilippo Syndrome, Sanfilippo A, Mucopolysaccharidosis III | REBISUFLIGENE ETISPARVOVEC | 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 trials1
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 |
|---|---|---|
| 32884151 | DERIVED | 1 |
Source PubMed
Data dates
The date of each source this release read.
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_0009655
GET https://conductscience.org/api/diseases/v1/MONDO_0009655/drugs