Disease · MONDO_0010674
mucopolysaccharidosis type 2
6 sourcesdiseases-2026-09-18-0.1.0
Cite this record
ConductScience Library. mucopolysaccharidosis type 2. Diseases, record MONDO_0010674. Release diseases-2026-09-18-0.1.0, 18 Sept 2026. https://conductscience.org/library/diseases/MONDO_0010674
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
Hunter syndrome, Hunter's syndrome, I2S deficiency, IDS deficiency, MPS 2, MPS II, MPS with skin involvement, MPS2, MPSII, Mucopolysaccharidosis Type II, SIDS deficiency, attenuated MPS (subtype; formerly known as mild MPS II), iduronate 2-sulfatase deficiency, mucopolysaccharidosis II, X-linked recessive, mucopolysaccharidosis with skin involvement, severe MPS II, sulfoiduronate sulfatase deficiency
- 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 |
|---|---|---|---|---|---|---|---|---|
| IDURSULFASE | CHEMBL1201826 | Enzyme | Approval | DailyMed and TTD | 13 | 10 | 8 | Drugs and targets |
| PABINAFUSP ALFA | CHEMBL4594565 | Protein | Approval | PMDA | 7 | 6 | 6 | Drugs and targets |
| IDURSULFASE BETA | CHEMBL4297988 | Enzyme | Approval | PMDA | 3 | 2 | 2 | Drugs and targets |
| TIVIDENOFUSP ALFA | CHEMBL4594537 | Protein | Phase 2/3 | ClinicalTrials.gov | 4 | 3 | 3 | Drugs and targets |
| SOMATROPIN | CHEMBL1201621 | Protein | Phase 2/3 | ClinicalTrials.gov | 1 | 1 | 1 | Drugs and targets |
| ADALIMUMAB | CHEMBL1201580 | Antibody | Phase 1/2 | ClinicalTrials.gov | 2 | 2 | 2 | Drugs and targets |
| VERENAFUSP ALFA | CHEMBL6068315 | Protein | Phase 1 | ClinicalTrials.gov | 1 | 1 | 1 | Drugs and targets |
| CLEMIDSOGENE LANPARVOVEC | CHEMBL5314820 | Gene | Unknown | USAN | 1 | 0 | 0 | Drugs and targets |
Targets4
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 |
| TFR2 | Transferrin receptor | 1 | Transferrin receptor binding agent | Approval | https://conductscience.org/library/genes/7036 | Drugs and targets |
| TFRC | Transferrin receptor | 1 | Transferrin receptor binding agent | Approval | https://conductscience.org/library/genes/7037 | Drugs and targets |
| TNF | Tumor necrosis factor | 1 | TNF-alpha inhibitor | Phase 1/2 | https://conductscience.org/library/genes/7124 | Drugs and targets |
Trials23
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. 2 more are not shown, since the checks do not confirm them.
| Trial | Title | Phase | Participants | Results first posted | Conditions | Drugs | Linked by | Source |
|---|---|---|---|---|---|---|---|---|
| NCT00069641 | Iduronate-2-sulfatase Enzyme Replacement Therapy in Mucopolysaccharidosis II (MPS II) | Phase 2/3 | 96 | 2015-05-13 | Mucopolysaccharidosis II | IDURSULFASE | Its record names the disease | ClinicalTrials.gov |
| NCT02412787 | Study of Long Term Safety and Clinical Outcomes of Idursulfase IT and Elaprase Treatment in Pediatric Participants Who Have Completed Study HGT-HIT-094 | Phase 2/3 | 56 | 2025-06-19 | Hunter Syndrome | IDURSULFASE | Its record names the disease | ClinicalTrials.gov |
| NCT00607386 | Safety and Clinical Outcomes in Hunter Syndrome Patients 5 Years of Age and Younger Receiving Idursulfase Therapy | Phase 4 | 28 | 2013-11-07 | Hunter Syndrome, Mucopolysaccharidosis II, MPS II | IDURSULFASE | Its record names the disease | ClinicalTrials.gov |
| NCT05058391 | A Study of Elaprase in Children and Adults With Hunter Syndrome (Mucopolysaccharidosis II) in India | Phase 4 | 5 | 2025-01-23 | Hunter Syndrome | IDURSULFASE | Its record names the disease | 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 |
| NCT06075537 | An Extension Study of the Long-Term Safety, Tolerability, and Efficacy of Tividenofusp Alfa (DNL310) in Participants With Mucopolysaccharidosis Type II (MPS II) From Study DNLI-E-0002 or Study DNLI-E-0007 | Phase 2/3 | 99 | Mucopolysaccharidosis II | TIVIDENOFUSP ALFA | Its record names the disease | ClinicalTrials.gov | |
| NCT04573023 | A Phase III Study of JR-141 in Patients With Mucopolysaccharidosis II (STARLIGHT) | Phase 3 | 86 | Mucopolysaccharidosis II | PABINAFUSP ALFA | Open Targets' reading of its record | ClinicalTrials.gov | |
| NCT05594992 | An Extension Study of JR-141 to Evaluate the Long-term Safety and Efficacy in MPS II (Hunter Syndrome) Subjects | Phase 3 | 80 | Mucopolysaccharidosis II | PABINAFUSP ALFA | Its record names the disease | ClinicalTrials.gov | |
| NCT05371613 | A Study to Determine the Efficacy and Safety of Tividenofusp Alfa (DNL310) vs Idursulfase in Pediatric and Young Adult Participants With Neuronopathic (nMPS II) or Non-Neuronopathic Mucopolysaccharidosis Type II (nnMPS II) | Phase 2/3 | 65 | Mucopolysaccharidosis II | TIVIDENOFUSP ALFA | Its record names the disease | ClinicalTrials.gov |
1 to 10 of 23
See all 23 trialsComparisons in its trials4
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 |
|---|---|---|---|---|---|
| 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 trials8
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_0010674
GET https://conductscience.org/api/diseases/v1/MONDO_0010674/trials?page=2