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
Research is ongoing for development of additional treatment options.
- Ueda M, Ando Y. Transl Neurodegener. 2014. doi:10.1186/2047-9158-3-19.
- Sekijima Y. J Neurol Neurosurg Psychiatry. 2015;86(9):1036-1043.
- Ando Y, Coelho T, Berk JL, et al. Orphanet J Rare Dis. 2013;8:31.