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ATTR-PN – a fatal disease that causes progressive and irreversible deterioration in neurological function4
ATTR-PN is a fatal hereditary disorder caused by genetic mutations resulting in the misfolding of TTR proteins, producing amyloid fibrils, which then accumulate primary in peripheral and autonomic nerves.4
>> The most common mutation associated with ATTR-PN is Val30met5

Patients with ATTR-PN often have damage to the autonomic nervous system, digestive tract, and other vital organs, sometimes including the heart.4

>> Nearly half of patients experience cardiac disease, which is a major cause of death5-8

Untreated patients have a life expectancy averaging 10 years from symptom onset with a progressive decline in neurologic function, ultimately resulting in death.5

>> Untreated patients with cardiac symptoms have a shorter median survival of ≈3.5 years once diagnosed, underscoring the importance of also monitoring patients for the symptoms of ATTR-CM in hereditary disease9
The stages of disease progression in patients with ATTR-PN
Note: Vyndaqel® is only indicated to treat adult patients with stage 1 ATTR-PN.)2