hATTR-PN

As we head into the Christmas season, I want to be mindful that not all of my readers celebrate the holiday. Additionally, I know not…

The National Organization for Rare Disorders (NORD) asks Americans to plan ahead to participate in the Light Up for Rare campaign to raise…

I am adamant about not letting the COVID-19 pandemic and the ongoing issues with my husband’s health dampen our Christmas spirit. This year, I…

Ionis Pharmaceuticals has entered into a collaboration agreement with AstraZeneca to develop and commercialize eplontersen, Ionis’ investigational therapy to treat transthyretin amyloidosis…

My husband Aubrey’s hereditary ATTR amyloidosis journey is included in a forthcoming book titled “Rare Disease Drug Development: Clinical, Scientific, Patient, and Caregiver…

Heart involvement is more prevalent among men with hereditary transthyretin (ATTRv) amyloidosis, a group of rare disorders that includes familial amyloid polyneuropathy (FAP), a study…

2021 has been a character-building year for me. I experienced challenges like never before, and I’m not just referring to the ones associated with the…

A clinical trial of the gene-editing therapy NTLA-2001 in people with familial amyloid polyneuropathy (FAP) is expanding to include those with a related condition, called ATTR…

I spent half the morning figuring out our plan for Christmas. In recent years, it’s been a tradition for the whole family — including pets…

Protego Biopharma has raised $51 million to support the development of therapies for diseases caused by protein misfolding, such as familial amyloid polyneuropathy…