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…
As anyone affected by a rare disease knows, treating the illness while trying to go about everyday life is an expensive undertaking. But exactly how expensive — in terms of direct and indirect costs across rare disease populations — might still come as a surprise: almost…
Nerve function deteriorates more rapidly in people with familial amyloid polyneuropathy (FAP) than in those with other peripheral nerve disorders, a literature review has found. These findings may improve the understanding of the natural history of nerve damage in FAP and…
Scientist Jonathan Wall, PhD, has won a $1.79-million grant to develop a potential treatment for amyloidosis, a group of conditions that includes familial amyloid polyneuropathy (FAP). The grant, from the National Institute of Diabetes and Digestive and Kidney Diseases, will fund a research project, titled “Developing a Theranostic…
Ionis Pharmaceuticals has launched a new grant program to fund novice researchers investigating transthyretin (ATTR) amyloidosis, a group of conditions that includes familial amyloid polyneuropathy (FAP), also known as hereditary ATTR. “We are proud to establish the Janice Wiesman Young Investigator Grant program, an important initiative to…