hATTR-PN

I have previously written about the importance of staying positive despite our circumstances. Whether you are a patient or a caregiver to a…

An elderly man in Germany was found to carry one copy of a new mutation in the TTR gene that caused both neurological and heart…

Quantitative sensory testing (QST), a tool for monitoring nerve function, may help to detect problems in small nerve fibers in people with late-onset familial…

Alnylam Pharmaceuticals has submitted an application to the U.S. Food and Drug Administration (FDA) asking the agency to approve Onpattro (patisiran) for the…

A surgical procedure called suture trabeculotomy (SLOT) ab interno successfully treated glaucoma in nearly half of the eyes of familial amyloid polyneuropathy (FAP) patients treated…

Novo Nordisk is moving forward on a global Phase 2 clinical trial evaluating its experimental treatment NNC6019 in people with hereditary transthyretin amyloidosis (ATTR)…

Glu89Lys, a rare gene mutation that can cause familial amyloid polyneuropathy (FAP) — and is associated with early onset disease and a mix of…

One-time treatment with the experimental gene-editing therapy NTLA-2001 led to substantial reductions in the levels of toxic transthyretin protein that were sustained for several months…

This column will be my 94th since starting “Sunrise, Sunset.” My caregiving experience has taken me through many ups and downs and countless…

Alnylam Pharmaceuticals will not conduct an interim analysis of its Phase 3 clinical trial testing Amvuttra (vutrisiran) in people with a…