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Hereditary transthyretin-mediated (hATTR) amyloidosis is a multisystem disease with heterogeneous symptom presentation1


Peripheral sensory-motor neuropathy
(e.g., neuropathic pain, paresthesia, weakness, bilateral carpal tunnel syndrome, difficulty walking)


Autonomic neuropathy
(e.g., orthostatic hypotension, recurrent
urinary tract infections, sexual dysfunction,
sweating abnormalities, urinary retention)


Gastrointestinal manifestations
(e.g., diarrhea, nausea, vomiting,
unintentional weight loss)


Cardiovascular manifestations
(e.g., arrhythmias, conduction abnormalities,
heart failure)

ONPATTRO® (patisiran) does not treat all of these symptoms.

  • More than 120 different transthyretin (TTR) gene mutations have been identified,
    with predominant symptom presentation varying by genotype2

    The most common mutations in the US are V122I, T60A, and V30M3

  • Some mutations are more common in certain populations, including those of Portuguese, Swedish, Japanese, African, and Irish descent4,5


  1. Conceição I, González-Duarte A, Obici L, et al. J Peripher Nerv Syst. 2016;21(1):5-9.
  2. Rowczenio DM, Noor I, Gillmore JD, et al. Human Mutat. 2014;35(9):E2403-E2412.
  3. Maurer MS, Hanna M, Grogan M, et al. J AM Coll Cardiol. 2016;68(2):161-172.
  4. Ando Y, Coelho T, Berk JL, et al. Orphanet J Rare Dis. 2013;8:31.
  5. Reilly MM, Staunton H, Harding AE. J Neurol Neurosurg Psych. 1995;59(1):45-49.