Dose-intensive therapy (DIT) for infantile Pompe disease: A pilot study

Keyphrases

• Pompe Disease 100%
• Intensive Care Medicine 100%
• Dose-intensive 100%
• RhGAA 100%
• Cross-reactive Immunologic Material 83%
• Enzyme Replacement Therapy 50%
• Clinical Outcomes 50%
• Antibody Formation 33%
• High Dose 33%
• Methotrexate 33%
• Toleration 33%
• Therapeutic Approaches 16%
• Monotherapy 16%
• Rituximab 16%
• Newborn Screening 16%
• Standard of Care 16%
• Severe Disease 16%
• Enzyme Activity 16%
• Patient Dose 16%
• Activities of Daily Living 16%
• Liver Transaminases 16%
• Antibody Level 16%
• Early Initiation 16%
• Therapy Regimen 16%
• Normal Activity 16%
• All Pediatric 16%
• Glucosidase 16%
• Developmental Milestones 16%
• Weekly Administration 16%
• Biventricular Hypertrophy 16%
• Recombinant Human Acid α-glucosidase 16%

Medicine and Dentistry

• Intensive Care 100%
• Glycogen Storage Disease Type II 100%
• Enzyme Replacement Therapy 60%
• Drug Megadose 40%
• Methotrexate 40%
• Pediatrics 20%
• Ventricular Hypertrophy 20%
• Monotherapy 20%
• Newborn Screening 20%
• Alglucosidase Alfa 20%
• Rituximab 20%
• Enzyme Activity 20%
• Transaminase 20%
• Developmental Milestone 20%
• Glucosidase 20%

Pharmacology, Toxicology and Pharmaceutical Science

• Glycogen Storage Disease Type 2 100%
• Replacement Therapy 60%
• Methotrexate 40%
• Heart Ventricle Hypertrophy 20%
• Rituximab 20%
• Alglucosidase Alfa 20%
• Aminotransferase 20%
• Glucosidase 20%
• Monotherapy 20%