Human disease
mucopolysaccharidosis type 2
Mondo defines it as: “A lysosomal storage disease leading to a massive accumulation of glycosaminoglycans and a wide variety of symptoms including distinctive coarse facial features, short stature, cardio-respiratory involvement and skeletal abnormalities. It manifests as a continuum varying from a severe to an attenuated form without neuronal involvement.”
1 disease group in OMIA reaches this disease, through OMIA's own Mondo link or its OMIM numbers. The page shows the chain. It does not say whether an animal is a model of this disease.
Animal diseases
By natural-model score for this disease, highest first. The score says how complete and how well supported each chain is, by stated rules. How the score works.
| OMIA group | Name | Found by | Species | The chain reached | Score |
|---|---|---|---|---|---|
| 1317 | Mucopolysaccharidosis II | OMIA's OMIM numbers | 2 | Human disease |
37.0
|
Human genes
Each human gene of these disease groups that Open Targets links to this disease.
Open Targets links none of these groups' human genes to this disease.
Drugs tested for this disease
Each ChEMBL drug that has been tested for this disease, and the human genes above it acts on, if any. Open Targets gives some drugs, such as most enzyme replacement therapies, no target gene.
| Drug | Acts on | Latest stage for this disease |
|---|---|---|
| Idursulfase | None of these genes | Approved |
| Idursulfase Beta | None of these genes | Approved |
| Pabinafusp Alfa | None of these genes | Approved |
| Somatropin | None of these genes | Phase 2/3 |
| Tividenofusp Alfa | None of these genes | Phase 2/3 |
| Adalimumab | None of these genes | Phase 1/2 |
| Verenafusp Alfa | None of these genes | Phase 1 |
| Clemidsogene Lanparvovec | None of these genes | Unknown |
The drug data is ChEMBL's, under CC BY-SA 3.0, and came through Open Targets release 26.09.