Throughout 2024, Amyloidosis News Today informed our readers about the latest developments in treatment and clinical trials for familial amyloid polyneuropathy (FAP). Here…
News
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 single dose of nucresiran, an experimental therapy for familial amyloid polyneuropathy (FAP) and other forms of ATTR amyloidosis, safely resulted in significant and sustained…
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…
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…
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…
Diflunisal is used off-label to treat familial amyloid polyneuropathy (FAP), but it may be as safe and effective as tafamidis free acid, which is…
HATTR-PN
NewsFAP therapy Amvuttra closer to reimbursement in Canada
Adults with familial amyloid polyneuropathy (FAP) living in Canada may soon be able to receive Amvuttra (vutisiran) at little or no cost through…
The Medicines and Healthcare products Regulatory Agency (MHRA) in the U.K. has approved the injection therapy eplontersen — under the brand name Wainzua —…
Applying a fluorescent green tracer called AMDX-9101 during a routine eye exam may enable the detection of the toxic transthyretin protein deposits that cause symptoms…
Recent Posts
- Early nerve damage seen in woman with rare TTR mutation
- AInylam initiatives aim to speed ATTR-CM diagnosis, coordinate care
- Blood test for Alzheimer’s may help in making hATTR-PN diagnosis
- Women with ATTR-CM face gaps in treatment, new study reports
- Real-world study finds Amvuttra helps keep hATTR-PN, life quality stable