hATTR-PN

Precision BioScience’s gene-editing therapy for transthyretin amyloidosis (ATTR), a group of disorders that also encompasses familial amyloid polyneuropathy (FAP), shows therapeutic potential in a preclinical study. Data from the study will be presented by Jenny A. Greig, PhD, senior director of the Gene Therapy Program at the Perelman…

Editor’s note: The Amyloidosis News Today team is providing in-depth coverage of the 2021 Virtual AAN Annual Meeting, April 17–22. Go here to read more stories from the conference. Diagnosing familial amyloid polyneuropathy (FAP) in people without a family history of the disease is still challenging, even though most…

One activity I really enjoy doing is going for coffee dates with my girlfriends. This allows me to take a break from my normal routine and spend time with those in my support network. As a carer, having people to confide in and share life’s journey with is vitally important…

Editor’s note: The Amyloidosis News Today team is providing in-depth coverage of the 2021 Virtual AAN Annual Meeting, April 17–22. Go here to read the latest stories from the conference. A determined stage of polyneuropathy disability (PND) might not be a reliable indicator of neuropathy progression among people with…

Editor’s note: The Amyloidosis News Today team is providing in-depth coverage of the 2021 Virtual AAN Annual Meeting, April 17–22. Go here to read more stories from the conference. Treatment with Onpattro (patisiran) lessened signs of nervous system damage in people with familial amyloid polyneuropathy (FAP), new clinical…

More than half of the people with rare diseases and their caregivers, asked in a survey, were undecided or less than willing to be vaccinated for COVID-19 if a vaccine was approved under emergency use authorization instead of the routine process, the EveryLife Foundation for Rare Disease reports. These findings…

Editor’s note: The Amyloidosis News Today team is providing in-depth coverage of the 2021 Virtual AAN Annual Meeting, April 17–22. Go here to read the latest stories from the conference. Onpattro (patisiran) over two years continues to ease symptoms of polyneuropathy and to improve quality of life among people…

Editor’s note: The Amyloidosis News Today team is providing in-depth coverage of the 2021 Virtual AAN Annual Meeting, April 17–22. Go here to read the latest stories from the conference. Vutrisiran, an investigational therapy for familial amyloid polyneuropathy (FAP), safely and effectively eased neurologic impairments and improved…

Some life events create impressions that stay with us forever. Our experiences, encounters, and journeys mold our character and hone our personality, making each of us into who we are today. What makes or breaks us when we face trials may be influenced by the particular calamity, but much…

Editor’s note: The Amyloidosis News Today team is providing in-depth coverage of the 2021 Virtual AAN Annual Meeting, April 17–22. Go here to read the latest stories from the conference. Among adults with familial amyloid polyneuropathy (FAP) whose disease progressed following…