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Migalastat

CHEMBL110458 Phase 4 승인됨 Small molecule
Half-Life
—
Bioavailability
—
Protein Binding
—
Molecular Weight
163.2 g/mol
LogP
-2.3
Phase
4

This pharmacological chaperone drug is used to treat Fabry disease, a rare inherited condition where a deficient enzyme (alpha-galactosidase A) allows fatty substances to accumulate in cells, damaging the kidneys, heart, and nervous system. It works by binding to and stabilizing specific mutant forms of the deficient enzyme, allowing more of it to reach its destination inside cells where it can function.

분자량

163.1700 g/mol

LogP

-2.30

TPSA

93.00 Ų

리핀스키 5의 법칙

통과

Pharmacokinetics (PK)

Pharmacodynamics (PD)

2D 구조

SVG PNG

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SMILES

                    
OC[C@H]1NC[C@H](O)[C@@H](O)[C@H]1O

                

                

                
                

                    
InChI

                    
InChI=1S/C6H13NO4/c8-2-3-5(10)6(11)4(9)1-7-3/h3-11H,1-2H2/t3-,4+,5+,6-/m1/s1

                

                

                

                    
                    

                        
Molecular Formula

                        
C6H13NO4

                    

                    
                    

                        
HBD / HBA

                        
5 / 5

                    

                    
                    

                        
회전 가능 결합

                        
1

                    

                    
                    
                    

                        
무거운 원자

                        
11

                    

                    
                

            


            
            

                
                


    
    
        
    
    
    
No targets recorded

    
    
Target interaction data is not yet available for this drug.

    



                
            


            
            

                
                


    
    
        
    
    
    
No interactions recorded

    
    
Drug interaction data is not yet available for this compound.

    



                
            


            
            

                
                


    
    
        
    
    
    
No side effects recorded

    
    
Side effect data is not yet available for this drug.

    



                
            


            
            
            

                
자주 묻는 질문

                

                    
                    

                        
                        

                            
This pharmacological chaperone drug is used to treat Fabry disease, a rare inherited condition where a deficient enzyme (alpha-galactosidase A) allows fatty substances to accumulate in cells, damaging the kidneys, heart, and nervous system. It works by binding to and stabilizing specific mutant forms of the deficient enzyme, allowing more of it to reach its destination inside cells where it can function.

                        

                    

                    

                    

                    

                    

                    

                        
                        

                            
Yes, Migalastat is an approved drug. It has reached clinical phase 4. It is classified as a Small molecule.

                        

                    

                

            

            

            
            

            
            

            
            
{# References & Data Sources section for drug detail pages.
   Renders standard pharmacological database links plus the drug's data_sources field. #}


    
References & Data Sources

    
    
        
        
  • 
            
            
                ChEMBL — European Bioinformatics Institute (EBI).
                CHEMBL110458.
                Open-access bioactivity database.
            
        
    • 
        
        
        
  • 
            
            
                PubChem — National Center for Biotechnology Information (NCBI).
                CID 176077.
                Chemical information database.
            
        
    • 
        
        
        
        
    

    
Data aggregated from publicly available pharmacological databases. Last updated 2026-03-28.




        


        
        
    


    



    
의학적 면책조항

    

        This content is for educational and informational purposes only. It is not a substitute for professional medical advice, diagnosis, or treatment. Always consult a qualified healthcare provider before making medication decisions.
    

    

        Data sources:
        ChEMBL,
        PubChem,
        DailyMed.