Pompe disease / glycogenosis type II / acid maltase deficiency research hub

Effects and health economic aspects of enzyme therapy in children and adults with Pompe disease; Long-term follow-up of patients receiving commercially available Myozyme

This study looks at how well Myozyme, a medicine for Pompe disease, works in children and adults over a long time. It checks things like muscle strength, breathing, and how people feel in their daily lives to understand its full effects.