All results
2 results for single arm open label multicenter study evaluating long term safety tolerability and
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Clinical Trials
This study is an international, multi-center, study of Pompe disease patients that are currently receiving enzyme-replacement therapy (ERT). The purpose of…
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Clinical Trials
This is a Phase I, single ascending dose, first in human, open-label, non-controlled, dose-escalation trial that will investigate intravenous infusions of…