hATTR-PN

At its first virtual investor event, biotech company Centogene set a bold mission: to cure 100 rare diseases within the next decade. A…

I was in the car with my daughters earlier today, and as we turned onto a busy street, we saw cars already lining up to…

A single dose of Intellia Therapeutics’ investigational CRISPR/Cas9-based gene-editing therapy NTLA-2001 safely results in a rapid and strong reduction in the levels of…

Alnylam Pharmaceuticals’ application for the approval of vutrisiran, its investigational therapy for familial amyloid polyneuropathy (FAP), is being reviewed by the U.S.

Lately I’ve had to remind my husband that he is not his disease. Aubrey was diagnosed in 2013 with hereditary ATTR amyloidosis, and after…

To help patient advocacy leaders and their partners better understand how global health statistics codes — known as ICD codes — are assigned, updated, and…

Those who wish to gain practical tools for living optimally with rare diseases are encouraged to attend the annual Living Rare Living Stronger Patient…

By the time this column is published, nearly two weeks will have passed since the kick-off of the two-day New Zealand Patients, Carers & Healthcare…

Amyl Therapeutics has raised €18.3 million — about $22.2 million — to develop its ClariTY technology platform to advance therapeutic candidates for familial…

I often write about the challenges I face as a carer to a spouse afflicted with hereditary ATTR amyloidosis. I highlight the heavy impact…