Rare disease enzyme replacement therapy landscape, 2026 mid-year reference

Reference layout of the enzyme replacement therapy class in rare disease as of mid-2026. Includes approved assets across lysosomal storage disease, next-generation engineered-enzyme programs, gene therapy alternatives, and the live commercial questions including chronic-infusion burden and CNS access. Useful as a quick orientation for cross-functional teams entering the lysosomal storage disease conversation, with companion gated Briefs on adjacent rare-disease commercial dynamics.