The main objective of this observational study is to evaluate the long-term safety and effectiveness of DTX401 for at least 10 years after DTX401 administration.
Age: 2 years - 66+
Gender: All
We've found 2 total result s for "Glycogen Storage Disease Type I".
Open-label, ascending dose trial of ACTUS-101 administered intravenously.
Age: 18 years - 66+
Gender: All