| # | Drug |
|---|---|
| 1 | anethole trithione |
| 2 | doxecitine |
| 3 | doxribtimine |
| 4 | eliglustat |
| 5 | fosdenopterin |
| 6 | givosiran |
| 7 | glycerol phenylbutyrate |
| 8 | lonafarnib |
| 9 | lumasiran |
| 10 | migalastat |
| 11 | miglustat |
| 12 | nedosiran |
| 13 | nitisinone |
| 14 | phenylacetic acid |
| 15 | phenylbutanoic acid |
| 16 | sapropterin |
| 17 | sepiapterin |
| 18 | sodium benzoate |
| 19 | teduglutide |
| 20 | thioctic acid |
| 21 | trientine |
| 22 | triheptanoin |
| 23 | uridine triacetate |
| 24 | zinc acetate |