Keyphrases
Gene Therapy
100%
Enzyme Replacement Therapy
100%
Pompe Disease
100%
Disease Genes
100%
Clinical Trials
40%
Natural History
40%
Early Onset
40%
Clinical Outcomes
40%
Recombinant Adeno-associated Virus (rAAV)
20%
Adulthood
20%
Cardiac Hypertrophy
20%
Clinical Response
20%
Disease Treatment
20%
Hypotonia
20%
Disease Spectrum
20%
Respiratory Failure
20%
Autosomal Recessive
20%
First Year of Life
20%
Severe Cases
20%
Symptom Onset
20%
Progressive Weakness
20%
Late-onset
20%
Lysosomal Enzymes
20%
Chronic Disability
20%
Disease Manifestations
20%
Mild Forms
20%
Glycogen Accumulation
20%
Metabolic Myopathy
20%
Pompe
20%
Cardiorespiratory Diseases
20%
Acid α-glucosidase
20%
Therapy Strategy
20%
Progressive Disability
20%
Medicine and Dentistry
Gene Therapy
100%
Enzyme Replacement Therapy
100%
Glycogen Storage Disease Type II
100%
Diseases
60%
Clinical Trial
40%
Ventricular Hypertrophy
20%
Adeno-Associated Virus
20%
Autosomal Recessive Inheritance
20%
Respiratory Failure
20%
Hypotonia
20%
Myopathy
20%
Lysosome Enzyme
20%
Weakness
20%
Cardiopulmonary Insufficiency
20%
Glucan 1,4 Alpha Glucosidase
20%
Pharmacology, Toxicology and Pharmaceutical Science
Replacement Therapy
100%
Glycogen Storage Disease Type 2
100%
Diseases
60%
Clinical Trial
40%
Preclinical Study
20%
Adeno Associated Virus
20%
Heart Ventricle Hypertrophy
20%
Weakness
20%
Myopathy
20%
Muscle Hypotonia
20%
Lysosome Enzyme
20%
Glucan 1,4 Alpha Glucosidase
20%
Cardiopulmonary Insufficiency
20%
Respiratory Failure
20%
Immunology and Microbiology
Preclinical Study
100%
Autosomal Recessive Inheritance
100%
Adeno Associated Virus
100%
Glucosidase
100%
Neuroscience
Enzyme Replacement Therapy
100%
Muscle Disorder
20%
Hypotonia
20%
Lysosome Enzyme
20%
Glucan 1,4 Alpha Glucosidase
20%
Adeno-Associated Virus
20%