Current & Investigational Treatment 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 treatment option that works by binding to the TTR protein preventing the dissociation into monomers.1,2

RNA interference (RNAi) therapeutics

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

Antisense oligonucleotides (ASOs)

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


Learn about an FDA-approved treatment option for the polyneuropathy of hATTR amyloidosis.

Research is ongoing for development of additional treatment options.

References:

  1. Ueda M, Ando Y. Transl Neurodegener. 2014. doi:10.1186/2047-9158-3-19.
  2. Sekijima Y. J Neurol Neurosurg Psychiatry. 2015;86(9):1036-1043.
  3. Ando Y, Coelho T, Berk JL, et al. Orphanet J Rare Dis. 2013;8:31.
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