Open-label, ascending dose trial of ACTUS-101 administered intravenously.
Age: 18 years - 66+
Gender: All
We've found 2 total result s for "Glycogen Storage Disease Type II".
The Pompe Registry is a global, multicenter, international, longitudinal, observational, and voluntary program for patients with Pompe disease, designed to track the disease's natural history and outcomes in patients, both treated and not. Data from the Registry are ...
Age: Birth - 66+
Gender: All