[HTML][HTML] Long-term monitoring of patients with infantile-onset Pompe disease on enzyme replacement therapy using a urinary glucose tetrasaccharide biomarker
SP Young, H Zhang, D Corzo, BL Thurberg, D Bali… - Genetics in …, 2009 - Elsevier
Purpose To investigate the correlation of the urinary glucose tetrasaccharide, Glcα1-6Glcα1-
4Glcα1-4Glc,(Glc 4) with skeletal muscle glycogen content and the long-term clinical
response to enzyme replacement therapy with recombinant human acid alpha glucosidase
in infantile Pompe disease. Methods Eighteen patients,≤ 6 months old, were enrolled in a
clinical trial of enzyme replacement therapy for up to 142 weeks. Urinary Glc 4, skeletal
muscle glycogen, and other clinical and laboratory assessments were made at baseline and …
4Glcα1-4Glc,(Glc 4) with skeletal muscle glycogen content and the long-term clinical
response to enzyme replacement therapy with recombinant human acid alpha glucosidase
in infantile Pompe disease. Methods Eighteen patients,≤ 6 months old, were enrolled in a
clinical trial of enzyme replacement therapy for up to 142 weeks. Urinary Glc 4, skeletal
muscle glycogen, and other clinical and laboratory assessments were made at baseline and …