News

Long-term treatment with Tegsedi (inotersen) reduced pain and improved physical functioning and health-related quality of life in patients with familial amyloid…

A diagnosis of familial amyloid polyneuropathy (FAP) due to a mutation in the TTR gene was made in an older man from China, a…

Larger studies are needed to back the use of nailfold capillaroscopy — a non-invasive technique for imaging tiny blood vessels in the fingers — for…

Alnylam Pharmaceuticals has selected Orsini Specialty Pharmacy to distribute Amvuttra (vutrisiran), which was recently approved by the U.S. Food and Drug Administration (FDA)…

A total of 30 patient advocacy groups working to further understanding and the needs of people living with rare diseases have been selected to receive…

Members of a Chinese family carrying a rare gene mutation causing familial amyloid polyneuropathy (FAP) experienced abnormal protein deposits in the eye — manifesting…

Nonprofits, scientists, governmental organizations, and the rare disease drug development industry have long cited 7,000 as the average…

A one-time treatment with NTLA-2001, Intellia Therapeutics’ experimental gene-editing therapy for people with familial amyloid polyneuropathy (FAP) and other types of ATTR amyloidosis, continued…

Several cases in which treatment with Onpattro (patisiran) was used to ease familial amyloid polyneuropathy (FAP) symptoms were described in a recent report…

Vutrisiran — now known by the brand name Amvuttra — has been approved by the U.S. Food and Drug Administration (FDA) to treat…