Safety, tolerability, pharmacokinetics, pharmacodynamics, and exploratory efficacy of the novel enzyme replacement therapy avalglucosidase alfa (neoGAA) in treatment-naïve and alglucosidase alfa-treated patients with late-onset Pompe disease: A phase 1, open-label, multicenter, multinational, ascending dose study

Medicin og biovidenskab

• Acids 6%
• Antibodies 4%
• Area Under Curve 6%
• Enzyme Replacement Therapy 75%
• Glucosidases 12%
• Glycogen 8%
• Glycogen Storage Disease Type II 83%
• human GAA protein 100%
• Late Onset Disorders 77%
• Lung 4%
• Pharmaceutical Preparations 3%
• Pharmacokinetics 43%
• Quadriceps Muscle 8%
• Safety 33%
• Therapeutics 9%
• Thorax 5%