Disease · MONDO_0009661
mucopolysaccharidosis type 6
6 sourcesdiseases-2026-09-18-0.1.0
Cite this record
ConductScience Library. mucopolysaccharidosis type 6. Diseases, record MONDO_0009661. Release diseases-2026-09-18-0.1.0, 18 Sept 2026. https://conductscience.org/library/diseases/MONDO_0009661
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
ARSB deficiency, ASB deficiency, MPS6, MPSVI, Maroteaux Lamy Syndrome, Maroteaux-Lamy disease, N-acetylgalactosamine 4-sulfatase deficiency, arylsulfatase B deficiency, mucopolysaccharidosis type VI, mucopolysaccharidosis type VI (Maroteaux-Lamy)
- Open Targets
Drugs8
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 |
|---|---|---|---|---|---|---|---|---|
| GALSULFASE | CHEMBL1201822 | Enzyme | Phase 3 | ClinicalTrials.gov | 4 | 4 | 4 | Drugs and targets |
| SOMATROPIN | CHEMBL1201621 | Protein | Phase 2/3 | ClinicalTrials.gov | 1 | 1 | 1 | 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 |
| ODIPARCIL | CHEMBL2107763 | Small molecule | Phase 2 | ClinicalTrials.gov | 1 | 1 | 1 | Drugs and targets |
| ADALIMUMAB | CHEMBL1201580 | Antibody | Phase 1/2 | ClinicalTrials.gov | 2 | 2 | 2 | Drugs and targets |
| VOSORITIDE | CHEMBL3707276 | Protein | Phase 1/2 | ClinicalTrials.gov | 1 | 1 | 1 | Drugs and targets |
Targets3
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 |
| NPR2 | Atrial natriuretic peptide receptor 2 | 1 | Atrial natriuretic peptide receptor B binding agent | Phase 1/2 | https://conductscience.org/library/genes/4882 | Drugs and targets |
| TNF | Tumor necrosis factor | 1 | TNF-alpha inhibitor | Phase 1/2 | https://conductscience.org/library/genes/7124 | Drugs and targets |
Trials10
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 |
| NCT00067470 | Study of Recombinant Human N-acetylgalactosamine 4-sulfatase (rhASB) in Patients With MPS VI | Phase 3 | 39 | 2009-08-17 | Mucopolysaccharidosis VI | GALSULFASE | Open Targets' reading of its record | ClinicalTrials.gov |
| NCT00104234 | Study of rhASB in Patients With Mucopolysaccharidosis VI | Phase 3 | 39 | 2009-03-06 | Mucopolysaccharidosis VI | GALSULFASE | Open Targets' reading of its record | 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 |
| NCT03370653 | A Study in MPS VI to Assess Safety and Efficacy of Odiparcil | Phase 2 | 20 | Mucopolysaccharidosis VI | ODIPARCIL | 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 | |
| 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 | |
| NCT00048620 | Study of Recombinant Human N-Acetylgalactosamine 4-Sulfatase in Patients With MPS VI | Phase 1 | Mucopolysaccharidosis VI | GALSULFASE | Open Targets' reading of its record | ClinicalTrials.gov | ||
| NCT00048711 | Open-Label Study of Efficacy and Safety of Recombinant Human N-acetylgalactosamine 4-sulfatase in Patients With MPS VI | Phase 2 | Mucopolysaccharidosis VI | GALSULFASE | Open Targets' reading of its record | ClinicalTrials.gov |
Comparisons in its trials5
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 |
|---|---|---|---|---|---|
| rhASB vs Placebo: serious adverse events | 3 of 19 | 4 of 20 | 39 | NCT00067470 | 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 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 |
|---|---|---|
| 33874971 | DERIVED | 2 |
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_0009661
GET https://conductscience.org/api/diseases/v1/MONDO_0009661/drugs