hATTR-PN

AT-01, an imaging agent being developed by Attralus, can help to differentiate between transthyretin (ATTR) amyloidosis — which includes familial amyloid polyneuropathy (FAP) — and other forms of amyloidosis, new clinical trial data indicate. The findings were presented in a poster, “Detection of Systemic AL…

It is a summer morning here in New Zealand. As I write this, a cool, gentle breeze blows through the house, and I am being serenaded by a symphony of native backyard birds. In moments like this, I am flooded with gratitude and peace. I bask in the knowledge that…

Familial amyloid polyneuropathy (FAP) is a progressive disorder that can lead to multiple health issues that affect your everyday life. Here are some ways in which family members can help support a person living with this rare disease. What is FAP? FAP patients experience severe neuropathy (damage to nerves)…

Vutrisiran, a second-generation RNA interference (RNAi) therapy candidate, safely and effectively reduces neurologic impairment and improves physical function and quality of life in adults with familial amyloid polyneuropathy (FAP). That’s according to top-line, nine-month data from the global HELIOS-A Phase 3 trial. Alnylam Pharmaceuticals, Vutrisiran’s developer, plans…

Sleep is an essential part of life that comes so naturally, from the moment we come into being. Yet sometimes we forget the impact that too much or too little sleep can have on our lives. I say “naturally,” but the truth is that falling asleep comes more easily for…

The National Organization for Rare Disorders (NORD) is seeking individuals willing to share real-life experiences with rare diseases to speak at its upcoming virtual Living Rare, Living Stronger NORD Patient and Family Forum. The interactive, patient-focused forum will be held online June 26-27. The deadline to apply for…

Benzbromarone (BBM), a medicine used to treat gout, can stabilize the TTR protein to the same degree as therapies used to treat people with familial amyloid polyneuropathy (FAP), a study showed. These findings support the further development of…

The case of a woman of Afro-Caribbean descent with early onset transthyretin (TTR) amyloidosis, who was found to carry a TTR mutation more commonly seen in those of Celtic descent, showed that this disorder can be more varied than previously known. Titled “Early-Onset of Transthyretin Amyloidosis in…

I can hear the shower going upstairs as I sit at my writing desk in my downstairs office. It is funny how the sound of running water always makes me feel a little melancholic and contemplative. At the moment, I am dwelling on a question someone posed to me after…

If you’ve been diagnosed with familial amyloid polyneuropathy (FAP), you may need to see a multidisciplinary treatment team of doctors — in addition to your primary care physician — so as to ensure that you get the best care. FAP is a progressive disorder that causes a buildup of…