Onasemnogene Abeparvovcec Infusion and Orfadin Suspension

• Generic Name: onasemnogene abeparvovec
• Dosage Forms: n.a.
• Other Brand Names: Zolgensma, Zolgensma

• It is used to treat spinal muscular atrophy.

• Generic Name: nitisinone
• Dosage Forms: capsule, oral suspension
• Other Brand Names: Orfadin, Nityr

Orfadin works by preventing the body from breaking down an amino acid called tyrosine and by keeping other toxic substances from building up and causing harm to your liver or kidneys.

Orfadin is used to treat a rare genetic condition called hereditary tyrosinemia type 1 (HT-1). HT-1 is a metabolic disorder that occurs when the body does not produce enough of an enzyme that breaks down proteins from certain foods. This condition occurs most often in young babies.

Orfadin may also be used for purposes not listed in this medication guide.