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MIGALASTAT
SynDRA canonical compound SYN0004048 — structure-anchored drug synonym record
| Clinical phase | Launched |
| Approval status | Approved · Rx |
| Primary target(s) | GLA |
| Mechanism of action | alpha-galactosidase a inhibitor |
| Indication | fabry disease |
| Disease area | endocrinology |
All known targets
- GLA
- AGL
- GAA
- GBA2
- GLB1
- Glb1
- LCT
- Lct
- MGAM
- SI
- aglC
- lacZ
Mechanisms of action (all sources)
- GLA STABILISER
- Alpha-galactosidase A stabiliser
- alpha-galactosidase a inhibitor
- GLA MODULATOR
- GLA PHARMACOLOGICAL CHAPERONE
Bioactivity evidence (ChEMBL activities table — not curator-asserted)
- GBA1
- MAOA
- HRH3
- HTR1A
- HTR2B
- KCNH2
- KDR
- MAN2B1
- MAN2B2
- MANBA
- OPRM1
- ADORA1
- PDE3A
- PDE4A
- PGR
- PTGS1
- SLC6A2
- SLC6A3
- SLC6A4
- TBXA2R
- GBA3
- ACHE
- GANAB
- FUCA1
- ADORA3
- ADRA1A
- ADRA2A
- ALOX15
- APEX1
- AR
- CHRM1
- CHRM2
- CYP1A2
- CYP2C19
- CYP2C9
- CYP2D6
- CYP3A4
- DRD1
- DRD3
- ESR1
Screening evidence (raw PubChem HTS — uncurated, known false-positive rate)
ATC classes
- A16A · OTHER ALIMENTARY TRACT AND METABOLISM PRODUCTS
Cross-references
Synonyms
- GALAFOLD
- Galafold
- migalastat HCl
- GR181413A
- 1-Deoxygalactonojirimycin hydrochloride
- 1-Deoxygalactonojirimycin
- migalastat hydrochloride
- galafold
- At-1001, gr-181314a
- DDIG
- D-Galactitol, 1,5-dideoxy-1,5-imino
- 1,5-Dideoxy-1,5-iminogalactitol
- 2-(Hydroxymethyl)-3,4,5-piperidinetriol hydrochloride
- C4XNY919FW
- 1-deoxygalactonojirimycin
- 1-Deoxygalactostatin
- migalastatum
- RefChem:56444
- 1-deoxygalacto-nojirimycin
- A16AX14
- DTXCID401507205
- NB-DGJ
- 1,5-Dideoxy-1,5-imino-D-galactitol
- Migalastat [INN]
- Migalastat [USAN:INN]
- UNII-C4XNY919FW
- DGJ
- NSC658776
- Tocris-1258
- MIGALASTAT [MI]
- Migalastat (USAN/INN)
- MIGALASTAT [USAN]
- MIGALASTAT [WHO-DD]
- D-galacto-1-Deoxynojirimycin
- orb1737999
- SCHEMBL29355275
- GTPL10200
- CHEBI:135923
Reported adverse events
- Abdominal pain
- Epistaxis
- Headache
- Nausea
- Back pain
- Cough
- Nasopharyngitis
- Pyrexia
- Urinary tract infection
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