Disease · MONDO_0009659
mucopolysaccharidosis type 4A
6 sourcesdiseases-2026-09-18-0.1.0
Cite this record
ConductScience Library. mucopolysaccharidosis type 4A. Diseases, record MONDO_0009659. Release diseases-2026-09-18-0.1.0, 18 Sept 2026. https://conductscience.org/library/diseases/MONDO_0009659
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
GALNS deficiency, MPS IV A, MPS4A, MPSIVA, Morquio disease type A, Morquio syndrome A, N-acetylgalactosamine-6-sulfate sulfatase deficiency, galactosamine-6-sulfatase deficiency, mucopolysaccharidosis type IVA
- Open Targets
Drugs2
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 |
|---|---|---|---|---|---|---|---|---|
| ELOSULFASE ALFA | CHEMBL2108676 | Enzyme | Approval | DailyMed and PMDA | 6 | 4 | 4 | Drugs and targets |
| VOSORITIDE | CHEMBL3707276 | Protein | 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 |
|---|---|---|---|---|---|
| NPR2 | Atrial natriuretic peptide receptor 2 | 1 | Atrial natriuretic peptide receptor B binding agent | Phase 1/2 | https://conductscience.org/library/genes/4882 |
Source Drugs and targets
Trials5
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 |
|---|---|---|---|---|---|---|---|---|
| NCT01609062 | Safety and Exercise Study of Two Doses of BMN 110 for Morquio A Syndrome | Phase 2 | 25 | 2016-02-01 | Mucopolysaccharidosis IVA, Morquio A Syndrome, MPS IVA | ELOSULFASE ALFA | Open Targets' reading of its record | ClinicalTrials.gov |
| NCT01242111 | A Study to Evaluate the Long-Term Efficacy and Safety of BMN 110 in Patients With Mucopolysaccharidosis IVA (Morquio A Syndrome) | Phase 1/2 | 20 | 2015-09-30 | MPS IV A, Mucopolysaccharidosis IVA, Morquio A Syndrome | ELOSULFASE ALFA | Its record names the disease | ClinicalTrials.gov |
| NCT01697319 | Efficacy and Safety Study of BMN 110 for Morquio A Syndrome Patients Who Have Limited Ambulation | Phase 2 | 16 | 2016-01-12 | Mucopolysaccharidosis IVA, Morquio A Syndrome, MPS IVA | ELOSULFASE ALFA | Open Targets' reading of its record | ClinicalTrials.gov |
| NCT01515956 | Study of BMN 110 in Pediatric Patients < 5 Years of Age With Mucopolysaccharidosis IVA (Morquio A Syndrome) | Phase 2 | 15 | 2017-07-06 | Mucopolysaccharidosis IVA, Morquio A Syndrome, MPS IVA | ELOSULFASE ALFA | Open Targets' reading of its record | ClinicalTrials.gov |
| NCT05845749 | Safety and Efficacy of Voxzogo for Growth Deficits in MPS IVA and VI | Phase 1/2 | 6 | MPS IVA, MPS VI | VOSORITIDE | Its record names the disease | ClinicalTrials.gov |
Comparisons in its trials1
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 |
|---|---|---|---|---|
| BMN 110 Weekly at 2.0 mg/kg/Week vs BMN 110 Weekly at 4.0 mg/kg/Week: serious adverse events | 3 of 15 | 2 of 10 | 25 | NCT01609062 |
Source 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.
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_0009659
GET https://conductscience.org/api/diseases/v1/MONDO_0009659/drugs