Alnylam Pharmaceuticals has initiated testing of a twice a year dosing regimen for vutrisiran, its investigational RNA-targeted therapy for familial amyloid polyneuropathy (FAP) and other forms of ATTR amyloidosis. The new dosing regime is being added to the open-label extension phase of the ongoing Phase 3 HELIOS-A trial (NCT03759379) that is now underway…
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 provided in-depth coverage of the 2021 Virtual AAN Annual Meeting, April 17–22. Go here to read the latest stories from the conference. There are roughly 2,700 people in the U.S. with hereditary transthyretin (ATTRv) amyloidosis, a group of rare disorders that also…
Note: This article was updated to include information from a Hopkins study published on May 5, 2021, of responses after a second vaccine dose was given to transplant patients. Long before COVID-19 changed the world, organ transplant recipients were wearing masks to shield themselves from airborne threats. Immunosuppressive medicines, which often…
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 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. Diagnosing familial amyloid polyneuropathy (FAP) in people without a family history of the disease is still challenging, even though most…
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…