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[38] A phase 3 clinical trial, started in January 2020 has an Estimated Study Completion Date of 30 June 2024 (NCT04232969)

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It is still a medical procedure that is administered intravenously

an orally delivered enzyme that finally reaches these hidden tissues could be its next chapter what I'd call an "ERT 3.0." Still preclinical, but a door well worth walking through

Clinical trials systematically document side effects experienced by participants