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Commercial
No data
Clinical
Clinical Trials
32 clinical trials
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Indications Phases 4
Indication | MeSH | Ontology | ICD-10 | Ph 1 | Ph 2 | Ph 3 | Ph 4 | Other | Total |
|---|---|---|---|---|---|---|---|---|---|
| Niemann-pick diseases | D009542 | — | E75.24 | — | 1 | — | 1 | — | 2 |
| Type a niemann-pick disease | D052536 | EFO_1001380 | E75.240 | — | 1 | — | 1 | — | 2 |
| Type c niemann-pick disease | D052556 | Orphanet_646 | E75.242 | — | 1 | — | 1 | — | 2 |
| Pick disease of the brain | D020774 | EFO_0003096 | G31.01 | — | — | — | 1 | — | 1 |
Indications Phases 3
Indication | MeSH | Ontology | ICD-10 | Ph 1 | Ph 2 | Ph 3 | Ph 4 | Other | Total |
|---|---|---|---|---|---|---|---|---|---|
| Cystic fibrosis | D003550 | EFO_0000390 | E84 | — | 3 | 1 | — | — | 3 |
| Gaucher disease | D005776 | Orphanet_355 | E75.22 | — | — | 1 | — | 1 | 2 |
| Tay-sachs disease | D013661 | Orphanet_845 | E75.02 | — | 1 | 1 | — | — | 2 |
| Gangliosidoses | D005733 | — | E75.10 | — | 1 | 1 | — | — | 2 |
| Gm2 gangliosidoses | D020143 | Orphanet_309152 | E75.0 | — | 1 | 1 | — | — | 2 |
| Sandhoff disease | D012497 | Orphanet_796 | E75.01 | — | — | 1 | — | — | 1 |
Indications Without Phase
No data
Epidemiology
Epidemiological information for investigational and approved indications
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Drug
General
| Drug common name | — |
| INN | — |
| Description | — |
| Classification | Unknown |
| Drug class | — |
| Image (chem structure or protein) |  |
| Structure (InChI/SMILES or Protein Sequence) | — |
Identifiers
| PDB | — |
| CAS-ID | — |
| RxCUI | — |
| ChEMBL ID | — |
| ChEBI ID | — |
| PubChem CID | — |
| DrugBank | — |
| UNII ID | — |
Target
No data
Variants
No data
Financial
No data
Trends
No data
Safety
Black-box Warning
No Black-box warning
Adverse Events
0 adverse events reported
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