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…
hATTR-PN
Amvuttra (vutrisiran) and Onpattro (patisiran) showed similar safety and efficacy for familial amyloid polyneuropathy (FAP) patients, according to new analyses from the Phase…
Treatment with Onpattro (patisiran) or Amvuttra (vutrisiran) leads to a reduction in blood levels of neurofilament light chain (NfL), a nerve cell damage…
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…
Changes in the nerve fibers of the cornea — the eye’s clear protective outer layer — may help in the early diagnosis of hereditary transthyretin…
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…
More than one year of treatment with eplontersen safely reduced symptoms of nerve damage and improved quality of life for adults with familial…
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…
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…
The U.S. Food and Drug Administration (FDA) has agreed to review an application from Ionis Pharmaceuticals seeking approval of eplontersen in treating …
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