Current & Investigational Treatment Options

There are multiple treatment options currently available for hATTR amyloidosis. Research is ongoing for the development of additional options.

Orthotopic liver transplant (OLT)

A potential treatment option for patients presenting with early stages of polyneuropathy that removes approximately 95% of the production of mutant TTR.1,2

TTR tetramer stabilizers

A drug molecule that binds to the TTR tetramer to prevent it from dissociating into monomers.1,2

RNA interference (RNAi) therapeutics

Double-stranded small interfering RNAs (siRNA) that bind to TTR messenger RNA (mRNA) and prevent production of TTR protein via the RNAi pathway.1-3

Antisense oligonucleotides (ASOs)

Short, chemically modified oligonucleotides that bind to TTR mRNA and prevent production of TTR protein via ASO-RNAse H-mediated cleavage.1-3

References:

  1. Sekijima Y. J Neurol Neurosurg Psychiatry. 2015;86(9):1036-1043.
  2. Ando Y, Coelho T, Berk JL, et al. Orphanet J Rare Dis. 2013;8:31.
  3. Ueda M, Ando Y. Transl Neurodegener. 2014;3:19.
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