hATTR-PN

Eurordis opened a campaign, called Rare 2030 Action, that is seeking to establish a European action plan for rare diseases to ensure that…

Enrollment is now completed in the Phase 3 APOLLO-B trial, which is assessing the safety and efficacy of Onpattro (patisiran) to treat heart disease…

Ahead of this year’s Rare Disease Week on Capitol Hill, held virtually July 14–22, the EveryLife Foundation will award grants to top advocates…

I count myself very fortunate to be in a position where I can speak to readers. Every week I sit at my desk, and in…

Our amyloidosis patient association here in New Zealand often holds routine catch-up sessions with the patients and carers that we support. I am a…

Attralus’ imaging agent AT-01 was able to detect amyloid deposits in the heart, even in patients without heart symptoms and with normal levels…

I will always remember May 12 as a special day. That’s because last week, I spent that day with my daughter doing something close to…

Alnylam Pharmaceuticals has initiated testing of a twice a year dosing regimen for vutrisiran, its investigational RNA-targeted therapy for familial amyloid polyneuropathy (FAP) and other forms of ATTR amyloidosis.

It has been a week since my husband, Aubrey, and I received our first doses of the COVID-19 vaccine. We feel fortunate to have…

Living with hereditary transthyretin amyloidosis (hATTR), a group of rare disorders that includes familial amyloid polyneuropathy (FAP), affects the quality of life for both…