Some rare inherited diseases are caused by a single missing enzyme, so the substance it should break down builds up in cells until organs fail. Enzyme replacement therapy supplies a manufactured version of the missing enzyme by regular infusion — usually every one or two weeks, for life. It does not cure the underlying genetic fault, but it can halt or slow the damage.
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Plain-English summaries and drug-class explainers are written and reviewed by the Priya Life Science editorial team, led by Sreepriya Prasannan (MSc Digital Transformation of Life Sciences (Innopharma Education / Griffith College); MSc & BSc Botany). Data is retrieved automatically from the sources above and cross-checked with AI-assisted verification (Anthropic's Claude) — brand and generic names are matched against the exact FDA product record so that a combination product or a different formulation cannot be mistaken for the drug on this page. An editor reviews the result before publication. We describe this in full in our editorial standards and corrections policy. The FDA data on this page was last retrieved on 26 Aug 2026.