Table 3.
Compounds that reduced the pathological phenotype of a zebrafish model of LGMD2I
| Therapeutic group/mechanism | Compound (PubChem CID) |
|---|---|
| antibiotics | ornidazole (28061) |
| sulfacetamide sodium hydrate (6419954) | |
| flumequine (3374) | |
| dihydrostreptomycin sulfate (6419912) | |
| metampicillin sodium salt (25195407) | |
| aztreonam (5742832) | |
| channel blockers/inhibitors | calycanthine (5392245) |
| ethosuximide (3291) | |
| kawain (5281565) | |
| dopamine antagonists | perphenazine (4748) |
| thioproperazine dimesylate (92178) | |
| α1 adrenergic receptor agonists | oxymetazoline hydrochloride (21114704) |
| metaraminol bitartrate (51371362) | |
| muscarinic antagonists | ipratropium bromide (11553369) |
| pirenzepine dihydrochloride (45114102) | |
| anti-neoplastics | betulin (72326) |
| paclitaxel (36314) | |
| smooth muscle relaxant | alverine citrate salt (21718) |
| skeletal muscle relaxant | chlorphensin carbamate (2724) |
| glucocorticoid | rimexolone (5311412) |
| triterpenoid nutraceutical | ursolic acid (64965) |
| metal chelator | pentetic acid (3053) |
| others † | citric acid (311) |
| triethanolamine (7618) |
Compounds identified by Serafini et al. [120]. Chemicals were screened at 2 dpf, 1 day after heat shock-inducible over-expression of a human FKRP transgene. By 5 dpf all compounds listed had reduced the percentage of larvae displaying the pathological phenotype (abnormal muscle birefringence, head and eye malformations, pericardiac edema) from approximately 50% to ≤10%.
†
Additional compounds screened because of their structural similarity to pentetic acid. Abbreviations: PDE, phosphodiesterase inhibitor.