Dosing & Uses
Hereditary Transthyretin Amyloidosis
Pending FDA approval for hereditary transthyretin-mediated amyloidosis polyneuropathy (hATTR-PN)
Pharmacology
Mechanism of Action
Antisense oligonucleotide that causes degradation of mutant and wild-type transthyretin (TTR) mRNA through binding to the TTR mRNA, which results in a reduction of serum TTR protein and TTR protein deposits in tissues
In patients with ATTR, TTR builds up as fibrils in tissues (eg, peripheral nerves, heart, GI system, eyes, kidneys, CNS, thyroid, bone marrow); the presence of TTR fibrils interferes with the normal functions of these tissues
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Formulary
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