![Treatment Options](/sites/default/files/2020-05/stroke-1_1.png)
![Treatment Options](/sites/default/files/2020-03/treatment.png)
Treatment Options
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There are several treatment options
for patients with hATTR
amyloidosis
There are several therapeutic approaches to
hereditary transthyretin-mediated (hATTR) amyloidosis
to consider.1 These treatment options address the pathogenesis of the disease in multiple ways:
![Liver](/sites/default/files/2020-03/liver.png)
Suppressing production of amyloidogenic transthyretin (TTR) in the liver
Antisense oligonucleotides (ASOs)1-3
Short, chemically modifed oligonucleotides that bind to
TTR messenger RNA (mRNA) and
prevent production of TTR protein
via RNAse H1-mediated cleavage.
RNA interference (RNAi) therapeutics1-3
Double-stranded small interfering RNAs (siRNA) that bind to TTR mRNA and prevent production of TTR protein via the RNA-induced silencing complex (RISC).
Orthotopic liver transplant (OLT)1,2
A potential treatment option for patients presenting with early stages of polyneuropathy that removes the
primary source of variant TTR.
![TTR tetramer](/sites/default/files/2020-03/ttr.png)
Stabilizing TTR tetramers
TTR tetramer stabilizers1,2
Drug molecule that binds to the TTR protein, preventing the dissociation into monomers.
Patient Resources
Family Resources
Information and Support Groups
References:
- 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.