hATTR-PN

Ionis Pharmaceuticals has entered into a collaboration agreement with AstraZeneca to develop and commercialize eplontersen, Ionis’ investigational therapy to treat transthyretin amyloidosis (ATTR). ATTR amyloidosis is a group of conditions characterized by the formation of toxic aggregates or clumps of the TTR protein that build up in…

My husband Aubrey’s hereditary ATTR amyloidosis journey is included in a forthcoming book titled “Rare Disease Drug Development: Clinical, Scientific, Patient, and Caregiver Perspectives.” He feels very touched that the editor saw fit to reach out to him. Emotional healing occurs when we can provide insight into a…

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 reports. Findings suggest that female sex may be protective against heart disease in ATTRv, and that being male may also contribute to disease worsening. The…

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 COVID-19 pandemic. This year, I suffered the loss of my mother, my beloved pet dog, and my sister-in-law. Just when I thought it couldn’t…

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 amyloidosis with cardiomyopathy (ATTR-CM), Intellia Therapeutics, which is developing the therapy, reported. FAP is a form of ATTR amyloidosis — a group of disorders…

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 and my children’s partners — to go away for the holidays. We would book a holiday home near the beach and spend a few days…

Protego Biopharma has raised $51 million to support the development of therapies for diseases caused by protein misfolding, such as familial amyloid polyneuropathy (FAP). The series A financing was co-led by Lightspeed Venture Partners, Vida Ventures, and MPM Capital. As part of the financing, representatives from these three…

This is my 48th column since I began this providential opportunity as a writer at Amyloidosis News Today. To be given the platform to share my journey as a caregiver to a husband who is afflicted with hATTR amyloidosis is humbling and cathartic. Life as a caregiver has its…

An annual decline in sensory nerve function — one greater than 25% — occurs as long as two years before the onset of familial amyloid polyneuropathy (FAP) in people carrying Val30Met, the most common disease-causing mutation in FAP, a study shows. Such drops in function, as measured by non-invasive…

Life experience plays an integral role in shaping our behavior and thinking. I have come a long way from being an unsettled 18-year-old to a wiser and more focused woman in her 50s. Looking back, I see how the ups and downs we face shape our individuality. We are…