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Treatment Options

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Title

There are several treatment options
for patients with hATTR
amyloidosis

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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:

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Suppressing production of amyloidogenic transthyretin (TTR) in the liver

Antisense oligonucleotides (ASOs)1-3

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

RNA interference (RNAi) therapeutics1-3

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

Orthotopic liver transplant (OLT)1,2

A potential treatment option for patients presenting with early stages of polyneuropathy that removes
the
primary source of mutant TTR.

Stabilizing TTR tetramers

TTR tetramer stabilizers1,2

Drug molecule that binds to the TTR protein, preventing the dissociation into monomers.

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References

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.