In this personal essay, Erin Poyant describes how hereditary transthyretin amyloidosis (ATTR) amyloidosis affected her family long before they understood what they were dealing with. Her father became seriously ill and was eventually found to have hereditary ATTR amyloidosis caused by the V122I genetic variant, but the diagnosis came too late to meaningfully change his outcome. After his death, Erin wrestled with the possibility of genetic testing, unsure what value it would have when treatment options still felt limited. When she later developed numbness in her hand and foot, she pursued testing and learned she carried the same variant. Even then, she faced barriers, including having to push her own primary care provider to take the testing seriously.

Now monitored as a symptomatic V122I carrier without a confirmed diagnosis, Erin writes about the emotional strain of living in uncertainty: aware of her risk, aware of symptoms, but still without clear answers about whether detectable disease is present or when treatment thresholds will be met. She describes this as living “in the between,” caught between gratitude for medical progress and frustration over what still cannot be known or done. Over time, that frustration became advocacy. She now uses her experience to raise awareness, support others facing similar uncertainty, and help create a stronger sense of community around hereditary amyloidosis, especially for people affected by the V122I variant.

Reference: Poyant E. From frustration to advocacy: our hereditary ATTR amyloidosis story. Amyloidosis Research Consortium. Published March 31, 2026. Accessed April 21, 2026. https://arci.org/from-frustration-to-advocacy-our-hereditary-attr-amyloidosis-story/

This phase 3 ATTRibute-CM trial evaluated acoramidis in patients with transthyretin amyloid cardiomyopathy (ATTR-CM) and found that it improved the overall primary hierarchical outcome compared with placebo over 30 months. More than 600 patients were included in the primary analysis, most of whom were older men with wild-type ATTR-CM and New York Heart Association class II or III symptoms. Acoramidis performed better than placebo on the trial’s four-part primary analysis, which prioritized death from any cause, cardiovascular-related hospitalization, change in N-terminal pro–B-type natriuretic peptide (NT-proBNP), and change in 6-minute walk distance. It also lowered the rate of cardiovascular hospitalizations and showed favorable effects on NT-proBNP, supporting a meaningful impact on disease progression and clinical status.

The trial also showed benefits across several key secondary outcomes. Compared with placebo, acoramidis led to less decline in 6-minute walk distance, better quality of life on the Kansas City Cardiomyopathy Questionnaire–Overall Summary, and higher serum transthyretin levels, which is consistent with strong TTR stabilization. Although the prespecified mortality analysis alone was not statistically significant, the overall results still favored acoramidis. The safety profile was also reassuring, with similar overall adverse event rates and fewer serious adverse events than placebo. Overall, the authors conclude that acoramidis is an effective and safe treatment option for patients with ATTR-CM, with benefits that extend beyond biomarkers to functional status and quality of life.

Reference: Gillmore JD, Judge DP, Cappelli F, et al. Efficacy and safety of acoramidis in transthyretin amyloid cardiomyopathy. N Engl J Med. 2024 Jan 11;390(2):132-142. doi:10.1056/NEJMoa2305434.

This review summarizes how treatment for cardiac amyloidosis has advanced across both transthyretin cardiac amyloidosis (ATTR-CM) and light-chain cardiac amyloidosis (AL-CM). For ATTR-CM, approved and emerging therapies now target different stages of disease biology: tetramer stabilizers such as tafamidis and acoramidis help prevent transthyretin breakdown; gene silencers such as vutrisiran and eplontersen reduce TTR production; gene-editing approaches such as NTLA-2001 aim for potentially durable one-time suppression of TTR; and fibril-depleting antibodies are being developed to help clear existing amyloid deposits from the heart. The review emphasizes that these newer therapies have improved outcomes and expanded options well beyond supportive care, although many investigational agents still need confirmation in large phase 3 trials.

For AL-CM, treatment remains centered on eliminating the abnormal plasma cell clone that produces toxic light chains, with daratumumab plus bortezomib, cyclophosphamide, and dexamethasone now established as standard first-line therapy. The review also highlights newer plasma cell–directed strategies, including CAR T-cell therapies and bispecific antibodies, as well as fibril-depleting antibodies designed to remove deposited amyloid. Overall, the authors conclude that cardiac amyloidosis is increasingly becoming a treatable chronic disease. However, major unmet needs remain, especially for patients with advanced disease. Future progress will depend on earlier diagnosis, better disease monitoring, comparative effectiveness research, and confirmation of whether fibril-depleting therapies can meaningfully improve long-term outcomes.

Reference: Nguyen O, Kamna D, Masri A. New therapies to treat cardiac amyloidosis. Curr Opin Cardiol. 2025 Mar 1;40(2):98-106. doi: 10.1097/HCO.0000000000001198.