Editor’s note: The FAP 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 FAP 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 FAP 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 FAP 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…
Editor’s note: The FAP 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…
Chinese patients with hereditary transthyretin (ATTR) amyloidosis, also known as familial amyloid polyneuropathy (FAP), have highly variable genetic and clinical profiles, a retrospective study has found. Yet, according to study findings, Val30Met remains the most common genetic mutation associated with the disease in China. The study “Hereditary…
The EveryLife Foundation for Rare Diseases is accepting applications for a scholarship program that aims to help adults with a rare disease pursue personal goals through training and education. For a second year, the #RAREis Scholarship Fund — supported by Horizon Therapeutics – will award 35 one-time scholarships, each…
Gastrointestinal (GI) symptoms, particularly unintentional weight loss, occur frequently in people with hereditary transthyretin amyloidosis, also known as familial amyloid polyneuropathy (FAP), according to data from an Italian study. Indeed, more than 80% of the patients in this study reported experiencing at least one GI symptom, a percentage well…
The European Commission (EC) has granted orphan drug status to Intellia Therapeutics’ NTLA-2001 gene-editing therapy for transthyretin amyloidosis (ATTR), a group of disorders that also encompasses familial amyloid polyneuropathy (FAP). Orphan drug status comes with benefits designed to ease a potential medicine’s path to market. “This news is…