Inborn errors of metabolism:
Indications for: AMVUTTRA
Polyneuropathy of hereditary transthyretin-mediated amyloidosis.
Give by SC inj in abdomen, thighs, or upper arms. 25mg once every 3 months.
Supplement with recommended daily Vit. A allowance. Refer for eye exam if ocular symptoms of Vit. A deficiency occur (eg, night blindness). Moderate or severe hepatic impairment, severe renal impairment, or ESRD: not studied. Elderly. Pregnancy. Nursing mothers.
Transthyretin-directed small interfering RNA.
Arthralgia, dyspnea, Vit. A decreased; inj site reactions, AV heart block.
Generic Drug Availability:
Single-dose prefilled syringe—1