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Picture a patient in your practice who is in their late 60s and begins experiencing increasing fatigue, shortness of breath, and difficulty keeping up with the everyday activities they once handled with ease. For years, a patient may move from appointment to appointment searching for answers, and may be treated for heart failure, atrial fibrillation or simply told they are “getting older.”1 Their physicians believe that to be true. However, this patient may have transthyretin-mediated cardiac amyloidosis, or ATTR-CM. For many individuals living with ATTR-CM, the journey to understanding what is happening can be long, frustrating and emotionally exhausting, not only for themselves, but also for their families, caregivers, and healthcare professionals (HCPs) who are watching their overall health and well-being decline over time.

Healthcare systems are navigating unprecedented demands, especially in rare disease, where existing resources often struggle to keep pace with real-time clinical needs and the ongoing shift toward value-based care. In this conversation, Duane Clark, General Manager, Rare Disease at Sanofi, and Robert Hopkin, MD, Clinical Geneticist at Cincinnati Children’s Hospital Medical Center, discuss how the field is changing and how the Sanofi 1RARE model has grown to help meet these changes.