See also:
Amvuttra
Amvuttra is used to treat polyneuropathy associated with hereditary transthyretin-mediated ...
Cerdelga
Cerdelga (eliglustat) is used to treat mild to moderate Type 1 Gaucher Disease. Includes Cerdelga ...
Aqneursa
Aqneursa information from Drugs.com, includes Aqneursa side effects, interactions and indications.
Crysvita
Crysvita is used for familial hypophosphatemia, osteomalacia, X-Linked Hypophosphatemia
Galafold
Galafold (migalastat) is used to treat Fabry disease. Includes Galafold side effects, interactions ...
Givlaari
Givlaari injection is used for acute hepatic porphyria (AHP) to reduce the number of acute attacks ...
Iqirvo
Iqirvo (elafibranor) is used to treat primary biliary cholangitis (PBC) in combination with ...
Kuvan
Kuvan is used to help the body activate the enzyme needed to control blood levels of phenylalanine ...