Human disease
muscular dystrophy
Mondo defines it as: “Muscular dystrophy (MD) refers to a group of more than 30 genetic diseases characterized by progressive weakness and degeneration of the skeletal muscles that control movement. Some forms of MD are seen in newborns, infants or children, while others have late-onset and may not appear until middle age or later. The disorders differ in terms of the distribution and extent of muscle weakness (some forms of MD also affect cardiac muscle), age of onset, rate of progression, and pattern of inheritance. The prognosis for people with MD varies according to the type and progression of the disorder. There is no specific treatment to stop or reverse any form of MD. Treatment is supportive and may include physical therapy, respiratory therapy, speech therapy, orthopedic appliances used for support, corrective orthopedic surgery, and medicationsincluding corticosteroids, anticonvulsants (seizure medications), immunosuppressants, and antibiotics. Some individuals may need assisted ventilation to treat respiratory muscle weaknessor a pacemaker for cardiac (heart)abnormalities.”
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 |
|---|---|---|---|---|---|
| 679 | Muscular dystrophy | The group's name | 7 | Target evidence |
58.0
|
Human genes
Each human gene of these disease groups that Open Targets links to this disease.
| Human gene | Score | Evidence |
|---|---|---|
| WWP1 | 0.01 | 8 |
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 |
|---|---|---|
| Casimersen | None of these genes | Approved |
| Deflazacort | None of these genes | Approved |
| Eteplirsen | None of these genes | Approved |
| Golodirsen | None of these genes | Approved |
| Viltolarsen | None of these genes | Approved |
| Ataluren | None of these genes | Phase 2 |
| Domagrozumab | None of these genes | Phase 2 |
| Efmitermant Alfa | None of these genes | Phase 2 |
| Sildenafil | None of these genes | Phase 2 |
| Tadalafil | None of these genes | Phase 2 |
| Trehalose | None of these genes | Phase 2 |
| Atyr-1940 | None of these genes | Phase 1/2 |
| Stamulumab | None of these genes | Phase 1/2 |
| Bortezomib | None of these genes | Phase 1 |
| (+)-Epicatechin | None of these genes | Phase 1 |
| Omigapil | None of these genes | Phase 1 |
| Sodium Nitrate | None of these genes | Phase 1 |
| Talfirastide | None of these genes | Phase 1 |
| Pargyline | None of these genes | Preclinical |
| Eplerenone | None of these genes | Unknown |
| Kifunensine | None of these genes | Unknown |
| Sevasemten | 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.