Template:Genetic agents

genetic disorder agents

cystic fibrosis agents

• ivacaftor
• lumacaftor
• cystinosis agents
• cysteamine

enzyme replacement therapy

• gaucher disease agents
  • glucocerebrosidase (enzyme replacement therapy)
  • imiglucerase, taliglucerase alfa, velaglucerase alfa

glucosylceramide synthase inhibitors (substrate restriction therapy)

• eliglustat, miglustat

lysosomal acid lipase deficiency agents

• sebelipase alfa

miscellaneous

• agalsidase beta, alglucosidase alfa, alpha1-proteinase inhibitor, elosulfase alfa, galsulfase, idursulfase, laronidase, pegademase

homocystinuria agents

• betaine

Huntington disease agents

• Tetrabenazine

• • Vesicular Monoamine Transporter 2 (VMAT2) Inhibitors
    • Deutetrabenazine, Tetrabenazine, Valbenazine

Monoclonal Antibodies

• Burosumab

Tyrosinemia Agents

• Nitisinone

Urea Cycle Disorder Agents

• Carglumic acid, Lactulose, Phenylbutyrate, Rifaximin, Sodium benzoate

Hematologic Agents

• Eculizumab, Emapalumab, Emicizumab, Lanadelumab, Ravulizumab