Hereditary transthyretin amyloidosis: a model of medical progress for a fatal disease

Adams D, et al. Nat Rev Neurol. 2019;15(7):387-404.

Breakthrough advances enhancing care in ATTR amyloid cardiomyopathy.

Porcari A, et al. Eur J Int Med. 2024. epub ahead of print.

Patisiran, an RNAi therapeutic, for hereditary transthyretin amyloidosis.

Adams D, et al. N Engl J Med. 2018;379(1):11-21.

Inotersen treatment for patients with hereditary transthyretin amyloidosis.

Benson MD, et al. N Engl J Med. 2018;379(1):22-31.

Efficacy and safety of vutrisiran for patients with hereditary transthyretin-mediated amyloidosis with polyneuropathy: a randomized clinical trial.

Adams D, et al. Amyloid. 2022;30(1):1-9.

Eplontersen for hereditary transthyretin amyloidosis with polyneuropathy.

Coelho T, et al. JAMA. 2023;330(15):1448-1458.

Tafamidis treatment for patients with transthyretin amyloid cardiomyopathy.

Maurer MS, et al. N Engl J Med. 2018;379(11):1007-1016.

Patient Advocacy Organizations and Resources

Amyloidosis Foundation

The mission of the foundation is to support patients and families while promoting research, education, and awareness.

Amyloidosis Research Consortium (ARC)

ARC is a nonprofit organization dedicated to driving advances in the awareness, science, and treatment of amyloid diseases. ARC’s outreach and education inform and empower patients, families, caregivers, physicians, and researchers.

Amyloidosis Support Groups (ASG)

ASG is a nonprofit corporation formed for the primary purpose of starting and maintaining support groups throughout the United States, dedicated to providing peer group support to patients, caregivers, families and friends of those touched by this life-threatening disease. 

National Organization for Rare Disorders (NORD)

NORD was the first national nonprofit to represent all individuals and families affected by rare disease. Today we’re the only organization working at the intersection of care, research, policy and community for all rare diseases.