A committee of the U.S. Food and Drug Administration (FDA) has endorsed Onpattro (patisiran) for the treatment of the ATTR amyloidosis with cardiomyopathy (ATTR-CM), or heart damage. Earlier this year, Alnylam Pharmaceuticals, which is developing Onpattro, submitted a supplemental new drug application (sNDA) to the federal agency…
Amvuttra (vutrisiran) provided significant clinical benefits in multiple measures of quality of life and physical function in people with familial amyloid polyneuropathy (FAP), according to published details of the HELIOS-A clinical trial. Benefits, which also included early gains in nutritional status, were most pronounced in those in the…
Scientists have developed a first patient-reported questionnaire on quality of life that’s specific to transthyretin amyloidosis (ATTR). Called the Transthyretin Amyloidosis – Quality of Life Questionnaire (ATTR-QOL), it was created with significant patient involvement and guidance from a multidisciplinary group of clinical specialists, led by scientists with the Amyloidosis…
A newly identified mutation in the TTR gene led to various neurological and heart symptoms of ATTR amyloidosis in three older adults in Korea, a case series reported. These patients, with an average age of 77, experienced an earlier onset of nerve symptoms affecting the extremities and the autonomic nervous…
Adults with hereditary transthyretin amyloidosis (hATTR), a group of disorders that includes familial amyloid polyneuropathy (FAP), lived longer after treatment with tafamidis versus undergoing a liver transplant, a study reported. However, patients treated with tafamidis experienced a faster decline in neurological and heart-related outcomes than did those who…
Caring for a person with hereditary transthyretin amyloidosis (hATTR), a group of conditions that includes familial amyloid polyneuropathy (FAP), has a considerable impact on caregivers’ health-related quality of life (HRQoL) and productivity, a survey reported. The findings were in the study, “A survey exploring caregiver burden…
Novo Nordisk is moving forward on a global Phase 2 clinical trial evaluating its experimental treatment NNC6019 in people with hereditary transthyretin amyloidosis (ATTR) affecting the heart. The company’s ATTR amyloidosis research program, recently acquired from Prothena, also includes NNC6019 as a treatment for familial amyloid…
Older age and worse disease were significantly associated with cognitive impairments in people with familial amyloid polyneuropathy (FAP) who underwent a liver transplant, a large study suggested. Researchers noted these cognitive findings are consistent with the natural history of the disease. “No distinct pattern was found for…
Eplontersen reduced transthyretin (TTR) protein levels, eased disease progression, and improved quality of life in adults with familial amyloid polyneuropathy (FAP), according to an eight-month interim analysis of the NEURO-TTRansform study. Based on these data, the investigational therapy’s co-developers Ionis Pharmaceuticals and AstraZeneca will seek regulatory approval…
Members of a Chinese family carrying a rare gene mutation causing familial amyloid polyneuropathy (FAP) experienced abnormal protein deposits in the eye — manifesting as “floaters” — before having any other noticeable disease symptoms, a case series reported. Such protein deposits can lead to functional abnormalities of the retina,…
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