Transthyretin-Associated Amyloidoses Outcomes Survey (THAOS) (Survey ID: Fx-R-001)

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Investigator: Jeffrey W. Ralph, MD
Sponsor: FoldRx Pharmaceuticals

Location(s): United States

Description

THAOS is a global, multi-center, longitudinal observational survey open to all patients with transthyretin-associated amyloidoses (ATTR), including ATTR-PN (polyneuropathy), ATTR-CM (cardiomyopathy) and wild-type ATTR-CM. It is open-ended with a minimum duration of 10 years. Patients will be followed as long as they are able to participate.

The principal aims of this outcome survey are to better understand and characterize the natural history of the disease by studying a large and heterogenous patient population. Survey data may be used to develop new treatment guidelines and recommendations, and to inform and educate clinicians about the management of this disease.

Familial amyloid polyneuropathy, also called transthyretin-related hereditary amyloidosis,transthyretin amyloidosis or Corino de Andrade's disease, is an autosomal dominant neurodegenerative disease. Usually manifesting itself between 20 and 40 years of age, it is characterized by pain, paresthesia, muscular weakness and autonomic dysfunction. In its terminal state, the kidneys and the heart are affected. Characterized by the systemic deposition of amyloidogenic variants of thetransthyretin protein, especially in the peripheral nervous system, causing a progressive sensory and motor polyneuropathy.