Treatment type

Enzyme Replacement

Treatments and trials using Enzyme Replacement. Filter by country, access level, or patient stories.

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Health Canada/FDA ApprovedUnited States Canada

New Weekly Enzyme Treatment for Arginase-1 Deficiency

Loargys (pegzilarginase-nbln) for arginase-1 deficiency (ARG1-D)

The FDA (accelerated, 23 Feb 2026) and Health Canada (full NOC, 29 May 2026) have labelled a weekly enzyme replacement for hyperargininemia in people 2 years…

Loargys (pegzilarginase) is a weekly enzyme treatment for arginase 1 deficiency in people 2 and older, used with a low-protein diet.

September 7, 2026

FDA ApprovedUnited States

New Weekly Infusion for Children with Hunter Syndrome

Avlayah (tividenofusp alfa-eknm) for neurologic Hunter syndrome (MPS II)

FDA accelerated approval (24 March 2026 letter; press 25 March 2026) for Avlayah (tividenofusp alfa-eknm) — a once-weekly IV enzyme replacement for neurologic…

Avlayah is a weekly infusion for the brain effects of Hunter syndrome in children of at least 5 kilograms.

September 7, 2026