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…
Treatment with Onpattro (patisiran) or Amvuttra (vutrisiran) leads to a reduction in blood levels of neurofilament light chain (NfL), a nerve cell damage marker, among people with familial amyloid polyneuropathy (FAP), according to a new analysis of clinical trial data. Findings suggest that patients with a greater…
People with hereditary transthyretin amyloidosis (hATTR) who are treated with a combination of therapies appear to have better health outcomes than those given only one type of medication. That’s according to new findings presented at the 75th American Academy of Neurology (AAN) annual meeting, being held April 22-27 in Boston…
Changes in the nerve fibers of the cornea — the eye’s clear protective outer layer — may help in the early diagnosis of hereditary transthyretin amyloidosis (hATTR), a group of disorders that includes familial amyloid polyneuropathy (FAP), a study shows. A noninvasive imaging method, called corneal confocal microscopy (CCM), identified…
In the U.S., one-third of people with hereditary transthyretin amyloidosis (hATTR) — a group of disorders that includes familial amyloid polyneuropathy (FAP) — are diagnosed with carpal tunnel syndrome years before a hATTR diagnosis, a study indicated. This finding suggests that a history of carpal tunnel, a condition that affects…
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…
Tegsedi (inotersen) slows the progression of symptoms, particularly muscle weakness and sensation loss, in people with familial amyloid polyneuropathy (FAP), according to a post-hoc analysis of the Phase 3 NEURO-TTR trial. “These results support previous evidence demonstrating the efficacy of [Tegsedi] in this patient population,” its researchers wrote. Post-hoc analyses…
A modified surgical procedure to implant a small filtration device — called Ex-PRESS — was effective at treating glaucoma in most people with hereditary transthyretin amyloidosis (hATTR), a group of disorders that includes familial amyloid polyneuropathy (FAP), a study shows. The surgery also led to a significant drop in the…
The U.S. Food and Drug Administration (FDA) has agreed to review an application from Ionis Pharmaceuticals seeking approval of eplontersen in treating familial amyloid polyneuropathy (FAP). The agency is expected to announce its decision by Dec. 22, according to Ionis, which is co-developing eplontersen alongside…
The U.S. Food and Drug Administration (FDA) has agreed to review an application from Alnylam Pharmaceuticals seeking the approval of Onpattro (patisiran) for the treatment of transthyretin-mediated (ATTR) amyloidosis with cardiomyopathy. According to the company, the FDA is expected to announce a decision by Oct. 8. Alnylam submitted…