News

Quality of life questionnaire specific to FAP, other ATTR forms created

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…

Advisory meeting about Onpattro for ATTR-CM set for September

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…

Sudden clearing of ATTR amyloidosis with cardiomyopathy seen in UK

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…

Onpattro shows benefits for 1.5 years in ATTR cardiomyopathy trial

Treatment with Onpattro (patisiran) for 1.5 years leads to sustained reductions in disability progression among people with ATTR amyloidosis with cardiomyopathy (ATTR-CM), or damage to the heart. That’s according to interim data from the ongoing open-label extension (OLE) portion of the Phase 3 APOLLO-B clinical trial (NCT03997383). That…