Table 4.
Small molecules, approved of under clinical development, for the treatment of LSDs.
| LSD | Compound | Strategy | Reference | Trial Phase |
|---|---|---|---|---|
| FD | Migalastat | PC | Approved [73] | |
| Lucerastat | SRT | NCT03425539 | III | |
| Venglustat | SRT | NCT02489344 | II | |
| GD | Venglustat | PC | NCT02843035 | II |
| Afegostat | PC | NCT00813865 NCT00446550 NCT00433147 | II | |
| Ambroxol | PC | NCT03950050 NCT04388969 | II | |
| Miglustat | SRT | Approved [74] | ||
| Eliglustat | SRT | Approved [75] | ||
| PD | Miglustat | PC | NCT02185651 NCT04327973 NCT04808505 | III |
| Duvoglustat | PC | NCT01380743 NCT00688597 NCT04327973 | II | |
| Gangliosidosis GM-2 | Venglustat | SRT | NCT04221451 | III |
| Pyrimethamina | PC | NCT01102686 | I/II | |
| MPS IV | Odiparcil | SRT | NCT03370653 | II |
| NPC | Miglustat | SRT | Approved [76] | |
| Arimoclomol | Induces HSP70 chaperone synthesis | NCT02612129 | III | |
| Ostat | Histone deacetylase inhibitor that increase mutant NPC1 protein levels | NCT02124083 | I/II |