ATTR: Think Beyond Idiopathic Neuropathy

Date/Time: Saturday, September 9, 2023 - 7:30 PM – 8:30 PM
Track: Satellite Symposia
Room: Salon I (5th Floor)
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Sponsored by AstraZeneca

Transthyretin amyloidosis (ATTR) is a systemic disease with a known underlying pathology of misfolded TTR protein resulting in the formation of fibrils that deposit in tissues and organs. Misfolding of the TTR protein occurs in response to the dissociation/instability of the TTR tetramer. The clinical manifestations due to amyloid fibril deposits are heterogeneous and can vary from patient to patient, but typically include a combination of polyneuropathy (PN) and/or cardiomyopathy (CM) symptoms, as well as a range of other symptoms (eg, gastrointestinal, musculoskeletal). However, the low disease prevalence (estimated 10,000-50,000 cases in the United States), clinical ambiguity and overlap of symptoms, and limited clinical awareness can result in years of undiagnosed disease progression prior to obtaining an accurate diagnosis. This can substantially impact the outcomes of patients with ATTR and the ability for HCPs and specialists to positively intervene.

The ability to identify and diagnose patients—particularly, early on in disease progression—with ATTR can substantially impact clinical outcomes as well as mortality. Treatment options exist for those with PN and/or CM, which make it possible to reduce, and in some cases halt, the progression of symptoms and its impact on quality of life (QoL). Systemic amyloidosis requires a multidisciplinary collaboration between the different medical specialists.

This symposium is intended for ATTR treaters and disease specialists (eg, neurologists) with the goal of highlighting key and critical components of the burden of disease and the patient journey, from raising clinical suspicion for ATTR, to incorporating a diagnostic algorithm for patient identification and consideration of therapeutic approaches to manage clinical manifestations associated with PN and CM. HCPs will learn about the range of clinical manifestations, including key “red flag” symptoms, that patients with ATTR experience and that the clinical profile can vary from patient to patient. Furthermore, HCPs will understand how important their ability to recognize and intervene on behalf of patients, early in disease progression, has the potential to result in more positive outcomes and QoL.


Francy Shu, MD

Neurology, Baylor Scott & White Medical Center