Familial amyloid polyneuropathy (FAP) patients who carry the Val30Met mutation may experience central nervous system involvement early in…
Articles by Patricia Inácio, PhD
Treatment with Tegsedi (inotersen) for more than three years continued to slow disease progression and improve the quality of…
Larger studies are needed to back the use of nailfold capillaroscopy — a non-invasive technique for imaging tiny blood vessels…
A total of 30 patient advocacy groups working to further understanding and the needs of people living with rare diseases…
Vutrisiran continues to show promise at lessening heart damage in adults with familial amyloid polyneuropathy (FAP) with cardiac involvement, according…
Fat accumulation in the soleus muscle, a large muscle of the lower leg, may be indicative of disease progression in…
Early signs of swallowing difficulties and carpal tunnel syndrome may serve as biomarkers for familial amyloid polyneuropathy (FAP) patients…
Switching to Onpattro (patisiran) following liver transplant and treatment with tafamidis lessened persistent disease progression in a patient with severe…
A skin biopsy may help speed up the diagnosis of familial amyloid polyneuropathy (FAP), including for asymptomatic people with…
The U.S. Food and Drug Administration (FDA) granted orphan drug status to eplontersen, an investigational therapy that seeks to…