Contact this trialFirst, we need to learn more about you.
Enzyme Replacement Therapy
Avalglucosidase Alfa for Pompe Disease
Recruiting2 awardsPhase 3
Hawthorne, New York
This trialwill assess safety & effectiveness of a drug for up to 4 years in people newly diagnosed with a rare genetic disorder. Visits every other week & potentially every week.
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Get in touch with the study teamWe’ll send your responses to the study team and help you to setup your first screening. This is not the start of informed consent. Screening happens between you and the trial.