Since 2008, Rare Disease Day — the last day of February — has brought together patients, caregivers, family members, friends, and advocates from around the…
News
Vutrisiran, an experimental therapy for familial amyloid polyneuropathy (FAP), continued to safely alleviate neurologic impairments and overall disability over 18 months of treatment,…
The U.S. Food and Drug Administration (FDA) granted orphan drug status to eplontersen, an investigational therapy that seeks to prevent the buildup of toxic…
The toxic transthyretin (TTR) clumps that form in people with familial amyloid polyneuropathy (FAP) caused by Val30Met, the most common disease-causing mutation, are structurally…
A man with familial amyloid polyneuropathy (FAP) developed eye problems years after he was found to have an uncommon disease-causing mutation called ATTRD18E, according…
MRI scans of pelvis and leg muscles may serve as a marker of progression and treatment response in people with hereditary transthyretin (ATTRv) amyloidosis, a…
The vagus nerve, the longest cranial nerve running from the brain through to the abdomen, is wider in people with familial amyloid polyneuropathy (FAP)…
HATTR-PN
NewsPrepare to Light Up Buildings for Rare Disease Day 2022
The National Organization for Rare Disorders (NORD) asks Americans to plan ahead to participate in the Light Up for Rare campaign to raise…
Ionis Pharmaceuticals has entered into a collaboration agreement with AstraZeneca to develop and commercialize eplontersen, Ionis’ investigational therapy to treat transthyretin amyloidosis…
Heart involvement is more prevalent among men with hereditary transthyretin (ATTRv) amyloidosis, a group of rare disorders that includes familial amyloid polyneuropathy (FAP), a study…
Recent Posts
- Amvuttra may ease symptoms and improve daily life in ATTR-CM
- Early nerve damage seen in woman with rare TTR mutation
- AInylam initiatives aim to speed ATTR-CM diagnosis, coordinate care
- Blood test for Alzheimer’s may help in making hATTR-PN diagnosis
- Women with ATTR-CM face gaps in treatment, new study reports