Browse medicines 46 total in our library

["What is Amvuttra?", "Amvuttra (vutrisiran) is an injectable siRNA \\\\u2013 GalNAc conjugate that...

["What is asfotase alfa?", "Asfotase alfa is an enzyme replacement medicine that is used to treat hy...

["What is burosumab?", "Burosumab is a monoclonal antibody that targets and blocks the activity of a...

["Uses for burosumab-twza", "Burosumab-twza injection is used to treat X-linked hypophosphatemia (lo...

["What is Cerdelga?", "Cerdelga reduces the formation of a certain protein in the body in people wit...

["What is Crysvita?", "Crysvita is a monoclonal antibody that targets and blocks the activity of a b...

["What is eliglustat?", "Eliglustat reduces the formation of a certain protein in the body in people...

["What is eplontersen?", "Eplontersen (Wainua) is used to treat polyneuropathy of hereditary transth...

["What is fosdenopterin?", "Fosdenopterin is used to reduce the risk of death in children with molyb...

["What is Galafold?", "Galafold works by binding to and stabilizing a certain enzyme in the body cal...

["What is Givlaari?", "Givlaari (givosiran) is a treatment for Acute Hepatic Porphyria (AHP) that he...

["What is givosiran?", "Givosiran is used to treat acute hepatic porphyria in adults.", "Acute hepat...

["What is inotersen?", "Inotersen (Tegsedi) is an RNA-targeting treatment that may be used to reduce...

["What is lonafarnib?", "Lonafarnib is for use in people with certain rare genetic conditions that c...

["What is lumasiran?", "Lumasiran is used to treat a type of hyperoxaluria (high levels of oxalate i...

["What is metreleptin?", "Metreleptin is used together with diet to treat complications caused by le...

["What is migalastat?", "Migalastat works by binding to and stabilizing a certain enzyme in the body...

["What is miglustat?", "Miglustat is used to treat mild to moderate type 1 Gaucher disease in adults...