Vutrisiran, an investigational therapy for familial amyloid polyneuropathy (FAP), eased neurological impairments, lessened heart stress, and ameliorated quality of life, nutritional status, and social engagement in adult patients, according to new nine-month data from HELIOS-A trial. “These additional data from the HELIOS-A study show the potential of vutrisiran…
Novo Nordisk has acquired Prothena’s clinical-stage antibody PRX004, an investigational therapy for people with hereditary transthyretin amyloidosis (ATTR), which also includes familial amyloid polyneuropathy (FAP), as well as other non-hereditary forms of the disease. While the company initially will focus on developing PRX004 for the treatment of ATTR…
Living with hereditary transthyretin amyloidosis (hATTR), a group of rare disorders that includes familial amyloid polyneuropathy (FAP), affects the quality of life for both patients and their families, a large questionnaire study in Italy found. Among the more common and practical consequences for…
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…
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…
Intellia Therapeutics anticipates reporting interim data this year from a Phase 1 study evaluating the safety and activity profile of NTLA-2001, its investigational gene-editing therapy to treat hereditary transthyretin amyloidosis with polyneuropathy, also known as…
Symptoms related to familial amyloid polyneuropathy (FAP) and other forms of hereditary transthyretin (ATTR) amyloidosis were found in African American and Hispanic or Latinx individuals who unknowingly carried a disease-related genetic variant revealed by genomic screening, according to a recent study.
Benzbromarone (BBM), a medicine used to treat gout, can stabilize the TTR protein to the same degree as therapies used to treat people with familial amyloid polyneuropathy (FAP), a study showed. These findings support the further development of…
Onpattro (patisiran) is more effective than Tegsedi (inotersen) at reducing neurological impairment and improving quality of life in people with familial amyloid polyneuropathy (FAP), a comparison of data from two clinical trials reported. The…
Tegsedi (inotersen) continued to slow disease progression and improve the quality of life in people with familial amyloid polyneuropathy (FAP) after two years of treatment, and also maintained a positive safety profile up to five years, according to an extension…