Novel Advances in Hereditary Amyloid Transthyretin Amyloidosis: A Stakeholder Interchange Report

A recent stakeholder interchange convened by the Institute for Value-Based Medicine and The American Journal of Managed Care^® brought together thought leaders in both the clinical and managed care communities to discuss challenges, opportunities, and managed care implications for patients with hATTR amyloidosis. Participants discussed the general management of patients with rare diseases, reviewed the pathophysiology and challenges of treating patients with hATTR amyloidosis, and discussed managed care considerations when evaluating treatment options for patients with hATTR amyloidosis. This article is a summary of key points from the discussion with Sami L. Khella, MD, professor of clinical neurology at Penn Medicine and chief of neurology at Penn Presbyterian Medical Center, both in Philadelphia, Pennsylvania.

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