A report on a 64-year-old man with familial amyloid polyneuropathy (FAP) may be the first to describe the buildup of disease-causing toxic clumps called amyloid deposits inside Schwann cells, specialized nervous system cells. Schwann cells wrap around nerve fibers, covering them with myelin, a fat-rich protective layer…
hATTR-PN
Throughout 2024, Amyloidosis News Today informed our readers about the latest developments in treatment and clinical trials for familial amyloid polyneuropathy (FAP). Here is a list of the top 10 most read stories we published in 2024, along with a brief summary. We look forward to continuing to…
December is a month of university graduation here in New Zealand. All the hard work, blood, sweat, and tears conclude in a cacophony of salutations and shouts of joy from family members. Jaime Christmas celebrates with her son Joshua at his college graduation ceremony this month. (Courtesy of Jaime…
Problems with memory and problem-solving in people with hereditary transthyretin amyloidosis (ATTRv), a group of diseases that includes familial amyloid polyneuropathy (FAP), associated with a thinning in specific brain regions, according to a small brain imaging study. “The reasons for cognitive involvement in ATTRv are still a subject of…
According to the Murdoch Children’s Research Institute, there are more than 7,000 rare genetic diseases in the world, mostly affecting children. Additionally, about 300 million people worldwide live with a rare disease, and most of these diseases have no approved treatment. These numbers underscore the urgent need for more research…
A single dose of nucresiran, an experimental therapy for familial amyloid polyneuropathy (FAP) and other forms of ATTR amyloidosis, safely resulted in significant and sustained reductions in blood levels of the disease-driving transthyretin (TTR) protein in healthy people. That’s according to interim results from an ongoing, placebo-controlled Phase 1…
A mutation of the TTR gene that’s been exclusively reported in Chinese people causes an eye condition called vitreous opacity that can be successfully managed with eye surgery, but eventually recurs in nearly all cases after a mean of five years, a study shows. After surgery, which is called…
As a columnist for Amyloidosis News Today since October 2020, I’ve written about various topics, all related to my experience caring for a spouse who had hereditary ATTR amyloidosis. My columns touch on the emotional and physical challenges I faced as a caregiver, the adjustments we…
One year after a single dose of Intellia Therapeutics’ experimental gene-editing therapy NTLA-2001, blood levels of the disease-driving transthyretin (TTR) protein dropped by a mean of 91% in people with familial amyloid polyneuropathy (FAP). Available data at two years showed sustained TTR level reductions, which were accompanied…
The U.S. Food and Drug Administration (FDA) has given orphan drug designation to AT-02, Attralus’ investigational candidate for the treatment of transthyretin amyloidosis (ATTR), a group of diseases that includes familial amyloid polyneuropathy (FAP). Orphan drug status supports the development of potential treatments for rare diseases, those…
Recent Posts
- A look at the emotional and psychological effects of diagnosis
- ATTR amyloidosis cases on the rise in the US as diagnostics improve
- Nerve ultrasound able to spot silent damage in hATTR, study finds
- Heart rhythm problems common in adults with FAP-causing mutation
- The emotional burden of receiving negative genetic test results