Approved Enzyme Replacement Therapy (ERT) Fabrazyme.
Updated on July 6, 2023.
Approved Enzyme Replacement Therapy (ERT) Elfabrio
Updated on July 6, 2023 .
Approved Enzyme Replacement Therapy (ERT) Replagal in many countries but not in the U.S.
Updated on November 1, 2020.
PTC is a global biopharmaceutical company that discovers, develops, and commercializes clinically differentiated medicines for children and adults living with serious diseases of high unmet need.
Updated on Sept 15, 2026.
Gene therapy offers the prospect of long-term and potentially curative benefits to patients with genetic or acquired diseases ....
Updated on November 22, 2020.
The re-administrable, non-myeloablative cell-based gene therapy for lysosomal storage disorders.
Updated on Sept 15, 2026.