Observational Registry

About Amicus Therapeutics

Amicus Therapeutics is a fully integrated, global biotech with a mission to address the vast unmet need in rare diseases, where 95% of over 10,000 known conditions lack approved treatments. The company has achieved commercial success with two approved therapies for Fabry and Pompe disease, validating its dual-technology platform. Its strategy centers on deepening its impact in core lysosomal disorders, expanding into adjacent rare diseases like FSGS, and maintaining a patient-centric culture that drives innovation and operational execution.

About Amicus Therapeutics

Amicus Therapeutics is a fully integrated, global biotech with a mission to address the vast unmet need in rare diseases, where 95% of over 10,000 known conditions lack approved treatments. The company has achieved commercial success with two approved therapies for Fabry and Pompe disease, validating its dual-technology platform. Its strategy centers on deepening its impact in core lysosomal disorders, expanding into adjacent rare diseases like FSGS, and maintaining a patient-centric culture that drives innovation and operational execution.

Therapeutic Areas