Table-I.
LSDs treatment options under clinical trials.
| Disease | Drug | Category | Clinical Trial Phase |
|---|---|---|---|
| Mucoploysaccharidosis-IIIA | Recombinant human Heparan-N-Sulfatase | ERT | Phase 2 |
| Mucopolysaccharidosis-IVA | Recombinant human N-acetylgalactosamine-6-sulfatase (BMN 110) | ERT | Phase 3 |
| Mucopolysaccharidosis-VII | Recombinant human β-glucuronidase (UX003) | ERT | Phase 3 |
| Metachtomatic Leukodystrophy | Recombinant human Arylsulfastase A | ERT | Phase 1/2 |
| Wolman Disease | Sebelipase (SBC-102) | ERT | Phase 3 |
| Alpha-mannosidosis | Lamazyme | ERT | Phase 2 |
| Niemann Pick C/D | N-Acetylcysteine | SRT | Phase 1/2 |
| Migulstat | SRT | Phase 2 | |
| 2-hydroxy-proyl-beta-cyclodextrin | SRT | Phase 2 |