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…
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…
The prevalence of familial amyloid polyneuropathy (FAP) in Taiwan in recent years is consistent with other mid-to-high prevalence countries, but survival rates are lower than in other nations, according to a recent study. From 2008-2020, FAP prevalence, or the total number of people living with the disease, in Taiwan ranged…
Onpattro (patisiran), a liver-directed gene-silencing therapy, successfully slowed progression of symptoms that emerged in two brothers with hereditary transthyretin amyloidosis (hATTR) decades after their disease was stabilized by liver transplants, a study showed. In the reemergence of disease symptoms, the brothers showed also brain and eye problems that were…
Intellia Therapeutics is planning to launch pivotal clinical trials of its gene-editing therapy NTLA-2001 in people with familial amyloid polyneuropathy (FAP) and ATTR amyloidosis with cardiomyopathy (ATTR-CM). A pivotal trial is one where the data are expected to support an application for a therapy’s regulatory approval. Intellia is…