A high throughput glucocerebrosidase assay using the natural substrate glucosylceramide

Treatment of Fabry Disease: Current and Emerging Strategies

(2011) Paula Rozenfeld et al.   CURRENT PHARMACEUTICAL BIOTECHNOLOGY

Pompe Disease: From New Views on Pathophysiology to Innovative Therapeutic Strategies

(2011) Giancarlo Parenti et al.   CURRENT PHARMACEUTICAL BIOTECHNOLOGY

Evaluation of 2-thioxo-2,3,5,6,7,8-hexahydropyrimido[4,5-d]pyrimidin-4(1H)-one analogues as GAA activators

(2010) Juan J. Marugan et al.   EUROPEAN JOURNAL OF MEDICINAL CHEMISTRY

Pharmacological small molecules for the treatment of lysosomal storage disorders

(2010) BE Smid et al.   EXPERT OPINION ON INVESTIGATIONAL DRUGS

The pharmacological chaperone isofagomine increases the activity of the Gaucher disease L444P mutant form of β-glucosidase

(2010) Richie Khanna et al.   FEBS Journal

Goal-oriented therapy with miglustat in Gaucher disease

(2009) Gregory M. Pastores et al.   CURRENT MEDICAL RESEARCH AND OPINION

Treating lysosomal storage diseases with pharmacological chaperones: from concept to clinics

(2009) Giancarlo Parenti   EMBO Molecular Medicine

Glycoengineered Acid α-Glucosidase With Improved Efficacy at Correcting the Metabolic Aberrations and Motor Function Deficits in a Mouse Model of Pompe Disease

(2009) Yunxiang Zhu et al.   MOLECULAR THERAPY

A sensitive and reproducible fluorescent-based HPLC assay to measure the activity of acid as well as neutral β-glucocerebrosidases

(2008) Yasuhiro Hayashi et al.   ANALYTICAL BIOCHEMISTRY

Optimization and Validation of Two Miniaturized Glucocerebrosidase Enzyme Assays for High Throughput Screening

(2008) Daniel Urban et al.   COMBINATORIAL CHEMISTRY & HIGH THROUGHPUT SCREENING