Transthyretin amyloidosis (ATTR) is a complex, rapidly progressive, and ultimately fatal disease that effects many organs of the body, notably the heart and nervous system. Non-specific symptoms and limited disease awareness often delay diagnosis of ATTR; on average, diagnosis occurs more than 3 years after initial symptom onset. This delay can have devastating consequences. The median survival from time of diagnosis is less than 4 years for ATTR patients with cardiac involvement.
Progression of ATTR causes significant morbidity, impacts productivity and quality of life, and creates a significant economic burden the costs associated with progressively greater patient needs for supportive care. ATTR also disproportionately impacts certain patient populations—for example, of all Black Americans carry a V122I TTR mutation that substantially increases their likelihood of developing .
treatment options currently that address the underlying causes of ATTR, and commonly prescribed therapies have limited efficacy in ATTR. There is great urgency to meet the needs of individuals with ATTR, particularly when it comes to earlier diagnosis and additional treatment options in order to quality of life and time to spend with loved ones.