Hereditary ATTR Amyloidosis

In this ARC Talks special presentation, Coffee with ARC, some members of the ARC team will provide an overview of ARC's history and areas of focus, as well as our plans for 2022 and beyond. Original presentation date December 9, 2021

In amyloidosis, common symptoms such as gastrointestinal manifestations and neuropathy are often the most troublesome for patients. In our ARC Talks Webinar for patients and caregivers, amyloidosis experts from across multiple specialties explain approaches for symptom management. Our experts provide you with the knowledge you need to live well with amyloidosis. Original presentation date October 26, 2021

In this ARC Talks Webinar for patients and caregivers, Professor Julian Gillmore, the Head and Research Lead of the National Amyloidosis Centre in London, describes the role of gene editing in hereditary diseases like hATTR, and shares the early results of Intellia’s CRISPR trial. Intellia’s Head of Development for In Vivo programs, Dr. Liron Walsh, joins Professor Gillmore for a question and answer session.

We live in a time of rapid advances in genetic know-how and pharmacological technologies. The pace of discovery is accelerating, driving the development of new therapies, with 3 newly approved treatments. The exact course of hATTR varies with each patient, but the outlook holds promise for all. This booklet is designed as a comprehensive guide to help you and your family navigate treatment resources and options that would be most effective for you. Click the image above to open the booklet.

Hereditary transthyretin amyloidosis (hATTR) is a rare, systemic disease passed down through families. Caused by genetic mutations in the transthyretin (TTR) gene, it leads to a buildup of abnormal proteins called amyloid in one or more organs and tissues, impairing their function. Left untreated, hATTR can cause life-threatening complications. Early diagnosis and treatment are critical to prevent or delay progression of hATTR. Click the image above to open the booklet.

In this ARC Talks webinar, Dr. Kelsey Barrell from the University of Utah explains what causes neuropathy and other neurological symptoms in amyloidosis patients and offers suggestions to help manage these symptoms.

Nancy Verel, a nurse at the Cleveland Clinic, shares her story about her family's journey through her husband's AL amyloidosis diagnosis and treatment. Robert David from BMC Cancer Support Programs provides coping and support strategies for caregivers.

Despite having many of the "red flag" symptoms of amyloidosis, Cece still experienced a delay in diagnosis. Cece shares her story, how a genetic condition has impacted her family, and how a heart transplant changed her life.  

In this patient webinar, Mayo Clinic's occupational therapist Sarah Dahlhauser, OTD, OTR/L, and physical therapist Sarah Boyd, PT, DPT, discuss exercise principles for maintaining mobility and function, and home modifications for improved safety for amyloidosis patients.

Genetic counselors Emily Brown and Katelyn Swade provide an overview of the science of genetic testing. They also discuss importance of genetic counseling for hereditary ATTR patients and their families.

This webinar covers access to innovative new therapies through expanded access programs with guest speakers Jennifer Miller, PhD, Assistant Professor at Yale University School of Medicine and Alison Bateman-House, PhD, MPH, Assistant Professor at NYU School of Medicine.