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…
The Amyloidosis Foundation again is marking Amyloidosis Awareness Month, observed each March, with a “Light the Night for Amyloidosis” campaign. The initiative seeks to call attention to amyloidosis, a group of diseases that includes familial amyloid polyneuropathy (FAP), a progressive disorder caused by the accumulation of amyloid fibrils…
Adults with hereditary transthyretin amyloidosis (hATTR), a group of disorders that includes familial amyloid polyneuropathy (FAP), lived longer after treatment with tafamidis versus undergoing a liver transplant, a study reported. However, patients treated with tafamidis experienced a faster decline in neurological and heart-related outcomes than did those who…
Most people with late-onset familial amyloid polyneuropathy (FAP) experience pain as a result of nerve damage, referred to as neuropathic pain, a recent study highlights. Findings suggest that neuropathic pain is linked to worse life quality, and many patients do not experience relief from this type of pain even…
Sudoscan, a commercial device that evaluates sweat gland function, might be a useful noninvasive tool to assess damage to small nerve fibers in people with familial amyloid polyneuropathy (FAP), a small study showed. Those small nerve fibers usually are affected in the early stages of the disease, leading to symptoms like…
Caring for a person with hereditary transthyretin amyloidosis (hATTR), a group of conditions that includes familial amyloid polyneuropathy (FAP), has a considerable impact on caregivers’ health-related quality of life (HRQoL) and productivity, a survey reported. The findings were in the study, “A survey exploring caregiver burden…
Tegsedi (inotersen) helped to stabilize familial amyloid polyneuropathy (FAP) symptoms in a 71-year-old man, but its use also was associated with a rare case of liver damage after one year of treatment. His liver enzymes, a marker of damage to the organ, rose well beyond the normal range, scientists reported.
England’s National Institute for Health and Care Excellence (NICE) has issued a draft guidance recommending Amvuttra (vutrisiran) be available through the country’s National Health Service (NHS) for treating familial amyloid polyneuropathy (FAP). Amvuttra is one of the first rare disease treatments to get the green light from NICE…