Systematic Structure-Activity Study on Potential Chaperone Lead Compounds for Acid α-Glucosidase

Molecular and cellular characterization of novel α-mannosidosis mutations

(2011) Elina Kuokkanen et al.   HUMAN MOLECULAR GENETICS

Structural Basis for Substrate Selectivity in Human Maltase-Glucoamylase and Sucrase-Isomaltase N-terminal Domains

(2010) Lyann Sim et al.   JOURNAL OF BIOLOGICAL CHEMISTRY

Effects of pH and Iminosugar Pharmacological Chaperones on Lysosomal Glycosidase Structure and Stability

(2009) Raquel L. Lieberman et al.   BIOCHEMISTRY

New Glucosidase Inhibitors from an Ayurvedic Herbal Treatment for Type 2 Diabetes: Structures and Inhibition of Human Intestinal Maltase-Glucoamylase with Compounds fromSalacia reticulata

(2009) Lyann Sim et al.   BIOCHEMISTRY

The pharmacological chaperone 1-deoxynojirimycin increases the activity and lysosomal trafficking of multiple mutant forms of acid alpha-glucosidase

(2009) John J. Flanagan et al.   HUMAN MUTATION

Enzyme replacement therapy with alglucosidase alfa in 44 patients with late-onset glycogen storage disease type 2: 12-month results of an observational clinical trial

(2009) S. Strothotte et al.   JOURNAL OF NEUROLOGY

The Pharmacological Chaperone N-butyldeoxynojirimycin Enhances Enzyme Replacement Therapy in Pompe Disease Fibroblasts

(2009) Caterina Porto et al.   MOLECULAR THERAPY

Binding parameters and thermodynamics of the interaction of imino sugars with a recombinant human acid α-glucosidase (alglucosidase alfa): Insight into the complex formation mechanism

(2008) Michiru Yoshimizu et al.   CLINICA CHIMICA ACTA

Update of the Pompe disease mutation database with 107 sequence variants and a format for severity rating

(2008) Marian Kroos et al.   HUMAN MUTATION

Biochemical and pharmacological characterization of different recombinant acid α-glucosidase preparations evaluated for the treatment of Pompe disease

(2008) A.J. McVie-Wylie et al.   MOLECULAR GENETICS AND METABOLISM

Therapeutic approaches in glycogen storage disease type II/pompe disease

(2008) Benedikt Schoser et al.   Neurotherapeutics

A counterintuitive approach to treat enzyme deficiencies: use of enzyme inhibitors for restoring mutant enzyme activity

(2007) Jian-Qiang Fan   BIOLOGICAL CHEMISTRY

Human Intestinal Maltase–Glucoamylase: Crystal Structure of the N-Terminal Catalytic Subunit and Basis of Inhibition and Substrate Specificity

(2007) Lyann Sim et al.   JOURNAL OF MOLECULAR BIOLOGY