Publication | Open Access

Evaluation of N-nonyl-deoxygalactonojirimycin as a pharmacological chaperone for human GM1 gangliosidosis leads to identification of a feline model suitable for testing enzyme enhancement therapy

BiochemistryMedicineHuman Gm1 GangliosidosisGlycobiologyEnzyme Enhancement TherapyExperimental PharmacologyPharmacotherapyFeline ModelMetabolomicsDrug DevelopmentPharmacologyDrug DiscoveryDrug Resistance