Amyl Therapeutics has raised €18.3 million — about $22.2 million — to develop its ClariTY technology platform to advance therapeutic candidates for familial amyloid polyneuropathy (FAP) and other forms of amyloidosis. “The ClariTY platform offers the unique opportunity to generate therapeutic candidates able to target all amyloid fibril…
Eurordis opened a campaign, called Rare 2030 Action, that is seeking to establish a European action plan for rare diseases to ensure that none of the 30 million people in Europe living with rare diseases are left behind by the start of a new decade. As part of…
Enrollment is now completed in the Phase 3 APOLLO-B trial, which is assessing the safety and efficacy of Onpattro (patisiran) to treat heart disease in people with transthyretin-mediated (ATTR) amyloidosis, a group of conditions that includes familial amyloid polyneuropathy (FAP). The study (NCT03997383) has enrolled more than…
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…
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…
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…
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…
People living with hereditary transthyretin (hATTR) amyloidosis — which includes familial amyloid polyneuropathy (FAP) — report “profound” life changes due to their disease, particularly as it progresses and affects them physically, emotionally, and socially, a study based on patient interviews reported. These findings emphasize the need for better care…