hATTR-PN

A Russian military plane crash near Tetiana Zamorska’s home in Kyiv, Ukraine, was a sign that it was time for her and her family to leave. The treacherous, 34-hour pilgrimage that ultimately brought the group of eight by car to temporary accommodations in neighboring Poland last month was physically and emotionally difficult,…

For the first time since my husband, Aubrey, was diagnosed with hereditary ATTR amyloidosis in 2013, his care team brought up the subject of palliative care last week. Was it unexpected? Yes. Were we prepared for this new step? No. Despite the many challenges we have faced, both individually…

A genetic variant in TTR — the gene implicated in familial amyloid polyneuropathy (FAP) — was linked with a greater risk of heart failure and death among Black people living in the U.S. in a recent study. In fact, being a carrier of the specific…

As a caregiver to a spouse with hereditary ATTR amyloidosis, I’ve become familiar with our local hospital. The countless appointments and stays that Aubrey, my husband, has endured since diagnosis in 2013 have allowed me the opportunity to roam the building and acquaint myself with the layout of…

The European Commission is expected to propose a new governing framework for health data next month, called the European Health Data Space (EHDS), with the aim of connecting national health systems to facilitate secure and efficient transfer of data across systems in different European nations. The move is expected to…

Having a chronic illness is never easy, both for the sufferers and their caregivers. My name is Jaime Christmas, and my husband, Aubrey, started feeling unwell in late 2012. It all started with numbness in his fingertips, but it wasn’t until he lost his taste for food that he…

In a real-world study of patients with worse disease severity at the start of treatment, Tegsedi (inotersen) slowed the progression of hereditary transthyretin amyloidosis — a group of disorders that also includes familial amyloid polyneuropathy (FAP) — and preserved quality of life. As progression is associated with disease…

The National Organization for Rare Disorders (NORD) has updated its State Report Card to make it more digitally friendly and added telehealth to its categories of rare disease policy issues in a nod to its increased use during the ongoing COVID-19 pandemic. NORD’s report card project began seven…

As I write this, my husband is in the hospital due to complications of hereditary ATTR amyloidosis. Aubrey, 58, was diagnosed in 2013, and this nine-year journey has been transformative. We both have learned to adapt to an ever-changing health situation and roll with the times. In a past…

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 (FAP) developed by Alnylam Pharmaceuticals. A decision, originally anticipated this month, is now expected by July 14. According to Alnylam, the FDA recently inspected…