hATTR-PN

Patient registries are a hot topic of rare disease research and many organizations are taking advantage of this resource by signing up their patient communities…

Every week I write my column using my personal experience as a caregiver to a spouse diagnosed with hereditary ATTR amyloidosis. Writing demonstrates…

Early signs of swallowing difficulties and carpal tunnel syndrome may serve as biomarkers for familial amyloid polyneuropathy (FAP) patients carrying the Ala97Ser (A97S) mutation,…

As a columnist, I try to read and expose myself to topics that might inform my writing. Recently, I came across a phrase that piqued…

Horizon Therapeutics has launched its #RAREis Representation program aimed at increasing diversity, equity, and inclusion among patients with rare diseases. There are about 400 million…

A single dose of NTLA-2001, Intellia Therapeutics’ investigational gene-editing therapy, safely and effectively leads to rapid and sustained reductions in the levels of the…

Bionews, the publisher of this website, hosted a virtual panel discussion on Rare Disease Day 2022, taking a deeper dive into what it’s like…

I recently accompanied my husband, Aubrey, to an appointment with a specialist at the hospital and noticed a sign taped to the wall in the…

To mark Amyloidosis Awareness Month, observed each March, the Amyloidosis Foundation is reprising its “Light the Night for Amyloidosis” campaign. The month-long initiative is…

A rare disease puts an economic burden on the patients, families, and caregivers that it affects, and will no doubt be an integral part of…