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MIGLUSTAT
SynDRA canonical compound SYN0003282 — structure-anchored drug synonym record
| Clinical phase | Launched |
|---|
| Approval status | Approved · Rx/OTC |
|---|
| Primary target(s) | UGCG |
|---|
| Mechanism of action | Glycosyl transferase inhibitor |
|---|
| Indication | Gaucher disease |
|---|
| Disease area | hematology |
|---|
All known targets
- UGCG
- GAA
- AGL
- GBA2
- Gaa
- Gba2
- MGAM
- P0504H10.9
- SI
- SLC5A4
- Si
- Sis
Mechanisms of action (all sources)
- Glycosyl transferase inhibitor
- UGCG INHIBITOR
- Ceramide glucosyltransferase inhibitor
- MGAM INHIBITOR
Bioactivity evidence (ChEMBL activities table — not curator-asserted)
- ADORA1
- HRH2
- HTR1A
- HTR2A
- HTR2B
- HTR2C
- HTR3A
- KCNH2
- LCT
- LMNA
- MAOA
- MAPK1
- MC3R
- MLNR
- NPY1R
- NR1I2
- NR3C1
- OPRD1
- OPRK1
- OPRM1
- PDE3A
- PDE4D
- PGR
- POLI
- PTGS2
- SLC29A1
- SLC6A2
- SLC6A3
- SLC6A4
- HRH3
- HRH1
- ADORA2A
- GLB1
- ADORA3
- ADRA1A
- ADRA2A
- ADRA2B
- ADRA2C
- ADRB1
- ADRB2
Screening evidence (raw PubChem HTS — uncurated, known false-positive rate)
Drug classes
- Glucosylceramide Synthase Inhibitors
- Anti-HIV Agents
- Anti-Infective Agents
- Anti-Retroviral Agents
- Antiviral Agents
- Enzyme Inhibitors
- Glycoside Hydrolase Inhibitors
- Hypoglycemic Agents
- Glucosylceramide Synthase Inhibitor
ATC classes
- A16A · OTHER ALIMENTARY TRACT AND METABOLISM PRODUCTS
Cross-references
Synonyms
- OPFOLDA
- Opfolda
- YARGESA
- Yargesa
- ZAVESCA
- Zavesca
- N-Butyldeoxynojirimycin
- N-Butylmoranoline
- zavesca
- Butyldeoxynojirimycin
- N-butyldeoxynojirimycin
- OGT-918
- Miglustat dipharma
- Miglustat gen.orph
- BuDNJ
- Miglustatum
- NBV
- Vevesca
- Miglustat [USAN]
- SC-48334
- SC48334
- Miglustat, Hydrochloride
- N-Butyl-deoxynojirimycin
- N-Butyl-DNJ
- NB-DNJ
- Zavesca (TN)
- Miglustat (USAN/INN)
- N-Bu-DNJ
- N-(n-Butyl)deoxynojirimycin
- N-butyl-1-deoxynojirimycin
- N-(n-butyl)-1,5-dideoxy-1,5-imino-D-glucitol
- 1,5-Dideoxy-1,5-N-butylimino-D-glucitol
- 1,5-(BUTYLIMINO)-1,5-DIDEOXY-D-GLUCITOL
- OGT 918
- ADN3S497AZ
- SC 48334
- miglustatum
Reported adverse events
- Nausea
- Thrombocytopenia
- Tremor
- Abdominal distension
- Constipation
- Dizziness
- Dyspepsia
- Flatulence
- Headache
- Migraine
- Vomiting
- Weight
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