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…
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…
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…
Amvuttra (vutrisiran) and Onpattro (patisiran) showed similar safety and efficacy for familial amyloid polyneuropathy (FAP) patients, according to new analyses from the Phase 3 HELIOS-A trial that supported Amvuttra’s regulatory approval. Both therapies led to similar reductions in transthyretin, the protein that accumulates in FAP, and they were…
More than one year of treatment with eplontersen safely reduced symptoms of nerve damage and improved quality of life for adults with familial amyloid polyneuropathy (FAP), according to top-line data from the Phase 3 NEURO-TTRansform trial. The experimental therapy from Ionis Pharmaceuticals and AstraZeneca also significantly…
A surgical procedure called suture trabeculotomy (SLOT) ab interno successfully treated glaucoma in nearly half of the eyes of familial amyloid polyneuropathy (FAP) patients treated in a Japanese study, although its effectiveness declined over several years. Eight patients required additional surgery to help treat their glaucoma, a condition marked by…
A genetic variant in TTR — the gene implicated in familial amyloid polyneuropathy (FAP) — was linked with a greater risk of heart failure and death among Black people living in the U.S. in a recent study. In fact, being a carrier of the specific…