Miglustat 

CFTR corrector

Phase of research

FDA-/EMA-approved for Other Purpose

How it helps

Restore CFTR Function

General information

N/A

Synonyms

n-butyldeoxynojyrimicin, NB-DNJ

Marketed as

Dietary sources

N/A

Structure not available

C10H21NO4

Drug-Mutation Relation

results for 120del23 / F508del
See all data on Miglustat 

Treats

Mutation Link Tested on Impact factor Notes
F508del Airway Delivery of Low-Dose Miglustat Normalizes Nasal Potential Difference in F508del Cystic Fibrosis Mice mice 10.69
F508del Proteasome-Dependent Pharmacological Rescue of Cystic Fibrosis Transmembrane Conductance Regulator Revealed by Mutation of Glycine 622 human cell cultures 4.31

Does not treat

Mutation Link Tested on Impact factor Notes