Recombinant human acid alpha-glucosidase enzyme therapy for infantile glycogen storage disease type II: results of a phase I/II clinical trial.
Amalfitano, A
Recombinant human acid alpha-glucosidase enzyme therapy for infantile glycogen storage disease type II: results of a phase I/II clinical trial. [electronic resource] - Genetics in medicine : official journal of the American College of Medical Genetics - 132-8 p. digital
Publication Type: Clinical Trial; Clinical Trial, Phase I; Clinical Trial, Phase II; Journal Article; Research Support, Non-U.S. Gov't; Research Support, U.S. Gov't, P.H.S.
1098-3600
10.109700125817-200103000-00007 doi
Age Factors
Animals
Blotting, Western
CHO Cells
Cricetinae
Enzyme-Linked Immunosorbent Assay
Glycogen--metabolism
Glycogen Storage Disease Type II--therapy
Heart--physiology
Heart Diseases--genetics
Humans
Infant
Muscle, Skeletal--metabolism
Muscles--pathology
Myocardium--metabolism
Phenotype
Radiography, Thoracic
Recombinant Proteins--therapeutic use
Time Factors
X-Rays
alpha-Glucosidases--therapeutic use
Recombinant human acid alpha-glucosidase enzyme therapy for infantile glycogen storage disease type II: results of a phase I/II clinical trial. [electronic resource] - Genetics in medicine : official journal of the American College of Medical Genetics - 132-8 p. digital
Publication Type: Clinical Trial; Clinical Trial, Phase I; Clinical Trial, Phase II; Journal Article; Research Support, Non-U.S. Gov't; Research Support, U.S. Gov't, P.H.S.
1098-3600
10.109700125817-200103000-00007 doi
Age Factors
Animals
Blotting, Western
CHO Cells
Cricetinae
Enzyme-Linked Immunosorbent Assay
Glycogen--metabolism
Glycogen Storage Disease Type II--therapy
Heart--physiology
Heart Diseases--genetics
Humans
Infant
Muscle, Skeletal--metabolism
Muscles--pathology
Myocardium--metabolism
Phenotype
Radiography, Thoracic
Recombinant Proteins--therapeutic use
Time Factors
X-Rays
alpha-Glucosidases--therapeutic use