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
- 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.
- Ueda M, Ando Y. Transl Neurodegener. 2014;3:19.