Treatment type
Treatments and trials using Enzyme Replacement, with the same country and access zoom as New Developments.
All New Developments
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…
The FDA (accelerated, 23 Feb 2026) and Health Canada (full NOC, 29 May 2026) have labelled a weekly enzyme…
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…
FDA accelerated approval (24 March 2026 letter; press 25 March 2026) for Avlayah (tividenofusp…