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…
A 63-year-old man in Japan with a large bulge in his right arm bicep, called “Popeye sign,” was diagnosed with late-onset familial amyloid polyneuropathy (FAP), according to a case report which suggests this sign has potential to diagnose the disease. The reason behind the symptom remains unclear in this…
AstraZeneca has added Latin America to the list of regions in which it has exclusive commercialization rights for eplontersen, an investigational therapy being reviewed for approval by the U.S. Food and Drug Administration (FDA) to treat familial amyloid polyneuropathy (FAP). Eplontersen’s original developer, Ionis…
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…
Eplontersen continues to safely and effectively ease symptoms and improve quality of life for people with familial amyloid polyneuropathy (FAP) who have been on the treatment for more than 1.5 years in the Phase 3 NEURO-TTRansform clinical trial. That’s according to top-line, 85-week results announced by…
Advanced MRI imaging of the sciatic nerve — a large bundle of nerve fibers running from the lower back down the length of the leg — may help to distinguish familial amyloid polyneuropathy (FAP) patients from healthy people, a study found. Such scans also may work to identify presymptomatic…
A U.S. Food and Drug Administration (FDA) advisory committee will meet Sept. 13 to discuss Alnylam Pharmaceuticals‘ application of Onpattro (patisiran) for treating ATTR amyloidosis with cardiomyopathy (ATTR-CM), according to the company. Data from the placebo-controlled Phase 3 APOLLO-B clinical trial (NCT03997383) will be reviewed at…
Machine learning, a form of artificial intelligence, may help doctors to identify people suspected of familial amyloid polyneuropathy (FAP) and who should undergo a genetic test for the disease, a study in Italy suggested. Machine learning uses algorithms to analyze data, learn from its analyses, and make a prediction…
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…
ATTR amyloidosis with cardiomyopathy (ATTR-CM), noting damage to the heart, is usually considered to be an irreversible condition. But in a first known reporting, the disease spontaneously resolved for three men in the U.K. Accompanying ATTR-CM’s resolution was the presence in the bloodstream of antibodies targeted against the insoluble transthyretin…