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Selected Product Category: Rare Diseases and Specialized Treatments


Myalept

Recombinant leptin analog


Luxturna

Gene therapy (AAV2)


Folotyn

Antifolate chemotherapy


Brineura

Enzyme replacement therapy


Blincyto

Bispecific T-cell engager


Ravicti

Nitrogen-scavenging agent


Zokinvy

Farnesyltransferase inhibitor


Danyelza

Anti-GD2 monoclonal antibody

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