Adults with familial amyloid polyneuropathy (FAP) living in Canada may soon be able to receive Amvuttra (vutisiran) at little or no cost through public drug plans, the therapy’s developer said. Amvuttra is approved in the country for adult FAP patients with stage 1 or stage 2 polyneuropathy or…
Onpattro (patisiran) may help familial amyloid polyneuropathy (FAP) patients who respond poorly to tafamidis meglumine stabilize or have reduced symptoms of nerve damage, called polyneuropathy. The findings are part of of a real-world study in France that analyzed data from 24 patients on tafamidis meglumine, approved as…
Wainua (eplontersen) is able to slow disease progression and improve life quality for people with familial amyloid polyneuropathy (FAP) regardless of nutritional status, sex, or certain genetic factors. That’s according to new subgroup analyses from the Phase 3 NEURO-TTRansform clinical trial (NCT04136184), whose top-line data supported Wainua’s…
The electrical signaling patterns associated with polyneuropathy, or damage to nerves outside the brain and spinal cord, vary in a first nerve conduction study given people in earlier stages of familial amyloid polyneuropathy (FAP). That’s according to a small study in Italy, which also found that signs of polyneuropathy…
A single infusion of NTLA-2001, an experimental gene-editing therapy, generally is safe and leads to a marked reduction in blood levels of the harmful transthyretin (TTR) protein in people with familial amyloid polyneuropathy (FAP) and ATTR amyloidosis with cardiomyopathy (ATTR-CM). These updated, interim data cover 65 of 72 patients…
A newly developed second-generation transthyretin (TTR) protein stabilizer may be more effective at treating forms of TTR amyloidosis (ATTR), including familial amyloid polyneuropathy (FAP), than one already tested in patients, scientists in Spain announced. Called PITB, the molecule was seen to be better than tolcapone — a TTR…
The U.S. Food and Drug Administration (FDA) has rejected Alnylam Pharmaceuticals‘ application seeking the expansion of its Onpattro (patisiran) label to include treatment of people with ATTR amyloidosis with cardiomyopathy (ATTR-CM), or heart damage. Based on the regulatory rejection, Alnylam has decided to no longer pursue an approval of…
Attralus’ AT-01 may be more sensitive than a standard imaging agent at detecting toxic transthyretin (TTR) protein clumps in the heart of people with hereditary ATTR amyloidosis, a group of conditions that includes familial amyloid polyneuropathy (FAP), according to new data from a clinical trial. The findings suggest that…
An international panel of patient advocates and healthcare providers has published the first set of recommendations outlining steps for the holistic care of people living with hereditary transthyretin-mediated amyloidosis (hATTR), a group of disorders that includes familial amyloid polyneuropathy (FAP). The guidelines focus not only on early diagnosis…
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…