💊 Thuốc A-Z 🧬 Đích tác dụng 🛠 Công cụ 📚 Hướng dẫn
Tất cả thuốc Phân loại thuốc Lĩnh vực điều trị Tác dụng phụ
Hướng dẫn Từ điển thuật ngữ Dòng thời gian
So sánh Hành trình của thuốc Các họ thuốc
🇺🇸 English 🇰🇷 한국어 🇯🇵 日本語 🇨🇳 中文简体 🇪🇸 Español 🇧🇷 Português 🇮🇳 हिन्दी 🇸🇦 العربية 🇫🇷 Français 🇷🇺 Русский 🇩🇪 Deutsch 🇹🇷 Türkçe 🇻🇳 Tiếng Việt 🇮🇩 Bahasa Indonesia 🇹🇭 ไทย

Risdiplam

CHEMBL4297528 Phase 4 Đã phê duyệt Small molecule
Half-Life
—
Bioavailability
—
Protein Binding
—
Molecular Weight
401.5 g/mol
LogP
0.5
Phase
4

Risdiplam is an orally bioavailable small molecule splicing modifier that corrects aberrant splicing of the SMN2 pre-mRNA by binding to and stabilizing the exon 7 splicing enhancer, increasing the production of full-length functional survival motor neuron (SMN) protein. It is approved for the treatment of spinal muscular atrophy (SMA) in pediatric and adult patients and is under investigation for other RNA splicing-related conditions.

Khối lượng phân tử

401,5000 g/mol

LogP

0,50

TPSA

78,10 Ų

Lipinski RO5

Đạt

Lĩnh vực điều trị

Non-Small Cell Lung Cancer

Cơ chế tác dụng

Delivers synthetic messenger RNA encoding a specific protein to host cells, where it is translated by ribosomes to produce the therapeutic protein without integrating into the host genome.

Pharmacokinetics (PK)

Pharmacodynamics (PD)

Cơ chế

Delivers synthetic messenger RNA encoding a specific protein to host cells, where it is translated by ribosomes to produce the therapeutic protein without integrating into the host genome.

Cấu trúc 2D

SVG PNG

Cite this structure


                        

                            
                            
                        

                    

                


                
                

                    

                    

                        
Embed this structure

                        

                        

                            
                            
                        

                    

                

                

                
                

                    
SMILES

                    
Cc1cn2nc(-c3cc(=O)n4cc(N5CCNC6(CC6)C5)ccc4n3)cc(C)c2n1

                

                

                
                

                    
InChI

                    
InChI=1S/C22H23N7O/c1-14-9-18(26-29-11-15(2)24-21(14)29)17-10-20(30)28-12-16(3-4-19(28)25-17)27-8-7-23-22(13-27)5-6-22/h3-4,9-12,23H,5-8,13H2,1-2H3

                

                

                

                    
                    

                        
Molecular Formula

                        
C22H23N7O

                    

                    
                    

                        
HBD / HBA

                        
1 / 6

                    

                    
                    

                        
Liên kết có thể quay

                        
2

                    

                    
                    
                    

                        
Nguyên tử nặng

                        
30

                    

                    
                

            


            
            

                
                


    
    
        
    
    
    
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.

    



                
            


            
            
            

                
Câu hỏi thường gặp

                

                    
                    

                        
                        

                            
Risdiplam is an orally bioavailable small molecule splicing modifier that corrects aberrant splicing of the SMN2 pre-mRNA by binding to and stabilizing the exon 7 splicing enhancer, increasing the production of full-length functional survival motor neuron (SMN) protein. It is approved for the treatment of spinal muscular atrophy (SMA) in pediatric and adult patients and is under investigation for other RNA splicing-related conditions.

                        

                    

                    

                    

                    
                    

                        
                        

                            
Delivers synthetic messenger RNA encoding a specific protein to host cells, where it is translated by ribosomes to produce the therapeutic protein without integrating into the host genome.

                        

                    

                    

                    

                    

                        
                        

                            
Yes, Risdiplam 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).
                CHEMBL4297528.
                Open-access bioactivity database.
            
        
    • 
        
        
        
  • 
            
            
                PubChem — National Center for Biotechnology Information (NCBI).
                CID 118513932.
                Chemical information database.
            
        
    • 
        
        
        
        
    

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




        


        
        
    


    



    
Tuyên bố miễn trách y tế

    

        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.