The purpose of this study is to collect, store, and study blood samples from people with Lysosomal Storage Diseases (LSDs). Some examples of LSDs include Gaucher disease, Fabry disease, Pompe disease, and Mucopolysaccharidosis I. The samples from this study will provide...
We've found 2 total results for "Pompe disease".
Open-label, ascending dose trial of ACTUS-101 administered intravenously.
Age: 18 years - 66+