People with rare disorders have a worse healthcare experience than those affected by chronic diseases, according to the results of an international survey conducted by Eurordis-Rare Diseases Europe. Indeed, rare disease patients overall give their healthcare experience a medium-low rating, of 2.5 on a scale of 1 to 5,…
Vutrisiran, a second-generation RNA interference (RNAi) therapy candidate, safely and effectively reduces neurologic impairment and improves physical function and quality of life in adults with familial amyloid polyneuropathy (FAP). That’s according to top-line, nine-month data from the global HELIOS-A Phase 3 trial. Alnylam Pharmaceuticals, Vutrisiran’s developer, plans…
The first wave of COVID-19 in Europe severely disrupted access to care and raised stress and anxiety in people with rare diseases, negatively affecting their health and well-being, according to a survey conducted by Eurordis-Rare Diseases Europe. “People living with rare diseases in Europe have found themselves caught as collateral…
Eidos Therapeutics announced that the Phase 3 clinical trial evaluating its lead candidate acoramidis (formerly known as AG10) in treating adults with transthyretin amyloidosis cardiomyopathy (ATTR-CM) is fully enrolled. Top-line results from part A of the trial, called ATTRibute-CM (NCT03860935), are expected in late 2021 or early 2022, and from…
A single dose of NTLA-2001, Intellia Therapeutics’ investigational gene editing therapy for transthyretin amyloidosis (ATTR), results in a sustained reduction in the levels of the damaging transthyretin (TTR) protein for at least one year, according to preclinical data. Based on these promising data in non-human primates,…
The characteristic toxic buildup of mutant transthyretin (TTR) protein clumps in familial amyloid polyneuropathy (FAP) affects the development of nerve cell fibers by interacting with regulators of a protein called actin, according to a study in a fruit fly model of the disease. The data suggested that…
Long-term treatment with Onpattro (patisiran) safely and effectively halts or reverses disease progression in people with familial amyloid polyneuropathy (FAP), final data from a two-year Phase 2 trial show. The findings were consistent with results from the Phase 3 APOLLO trial (NCT01960348), which supported Onpattro’s approval…
Researchers have reported the first case of familial amyloid polyneuropathy (FAP) with chronic dry cough — caused by a less common mutation — in a large Chinese family. The case report, “Familial amyloid polyneuropathy with chronic paroxysmal dry cough in Mainland China: A Chinese family with…
The U.S. Food and Drug Administration (FDA) has approved Alnylam Pharmaceuticals’ Onpattro (patisiran) as the first-ever treatment for patients in the U.S. with familial amyloid polyneuropathy (FAP). “Today’s historic approval marks the arrival of a first-of-its kind treatment option for a rare and devastating condition with limited treatment options,”…
Alnylam Pharmaceuticals’ investigative patisiran treatment improves overall health and prevents worsening of walking ability in patients with familial amyloid polyneuropathy (FAP), according to a new analysis from the APOLLO Phase 3 study. FAP, also known as hereditary ATTR (hATTR) amyloidosis, is caused by mutations of the transthyretin (TTR) gene…
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