Lucerastat, an Iminosugar for Substrate Reduction Therapy: Pharmacokinetics, Tolerability, and Safety in Subjects With Mild, Moderate, and Severe Renal Function Impairment

Lucerastat, an iminosugar with potential as substrate reduction therapy for glycolipid storage disorders: safety, tolerability, and pharmacokinetics in healthy subjects

(2017) N. Guérard et al.   Orphanet Journal of Rare Diseases

Treatment of Fabry’s Disease with the Pharmacologic Chaperone Migalastat

(2016) Dominique P. Germain et al.   NEW ENGLAND JOURNAL OF MEDICINE

Evaluation of miglustat as maintenance therapy after enzyme therapy in adults with stable type 1 Gaucher disease: a prospective, open-label non-inferiority study

(2012) Timothy M Cox et al.   Orphanet Journal of Rare Diseases

Fabry disease

(2010) Dominique P Germain   Orphanet Journal of Rare Diseases

Fabry disease

(2009) Raphael Schiffmann   PHARMACOLOGY & THERAPEUTICS

N-butyldeoxygalactonojirimycin reduces brain ganglioside and GM2 content in neonatal Sandhoff disease mice

(2007) Rena C. Baek et al.   NEUROCHEMISTRY INTERNATIONAL