hATTR-PN

The National Organization for Rare Disorders (NORD) has updated its State Report Card to make it more digitally friendly and added telehealth to…

As I write this, my husband is in the hospital due to complications of hereditary ATTR amyloidosis. Aubrey, 58, was diagnosed in 2013, and…

The U.S. Food and Drug Administration (FDA) is extending by three months its review timeline for vutrisiran, an experimental therapy for familial amyloid polyneuropathy…

Every so often, my husband, Aubrey, and I go through this exercise where we evaluate our circumstances in life and decide if we should shake…

Small vesicles in the blood contribute to the clumping or aggregation, and toxic accumulation of the transthyretin (TTR) protein in people with familial amyloid…

The day arrived when COVID-19 stepped into our home via our youngest daughter. She found out that a university classmate who’d sat next to her…

A highly sensitive tool has been developed to help identify patients with familial amyloid polyneuropathy (FAP). Using a compound score based on clinical symptoms…

Healthcare providers involved in diagnosing and treating rare diseases believe that increased physician education and collaboration with specialized facilities will have the greatest positive impact…

In 2018, Gov. Bill Haslam made Tennessee the first U.S. state to declare March as Amyloidosis Awareness Month. Since then, those diagnosed with…

Familial amyloid polyneuropathy (FAP) caused by Thr49Ser, a rare disease-causing mutation, was identified for the first time in three patients from Japan, as described…