News

Onpattro Approval Sought for ATTR Amyloidosis With Cardiomyopathy

Alnylam Pharmaceuticals has submitted an application to the U.S. Food and Drug Administration (FDA) asking the agency to approve Onpattro (patisiran) for the treatment of transthyretin-mediated (ATTR) amyloidosis with cardiomyopathy. Onpattro already is approved to treat familial amyloid polyneuropathy (FAP), a genetic disorder in which toxic tangles…

Less Invasive Glaucoma Surgery Can Be Effective in Short Term

A surgical procedure called suture trabeculotomy (SLOT) ab interno successfully treated glaucoma in nearly half of the eyes of familial amyloid polyneuropathy (FAP) patients treated in a Japanese study, although its effectiveness declined over several years. Eight patients required additional surgery to help treat their glaucoma, a condition marked by…

NTLA-2001 Shows Benefits for ATTR Patients With Heart Disease

One-time treatment with the experimental gene-editing therapy NTLA-2001 led to substantial reductions in the levels of toxic transthyretin protein that were sustained for several months in people with ATTR amyloidosis with cardiomyopathy (ATTR-CM). That is according to data shared by NTLA-2001’s co-developer, Intellia Therapeutics, at the American Heart Association…

Older Age, Worse Disease Predict Cognitive Issues With FAP: Study

Older age and worse disease were significantly associated with cognitive impairments in people with familial amyloid polyneuropathy (FAP) who underwent a liver transplant, a large study suggested. Researchers noted these cognitive findings are consistent with the natural history of the disease. “No distinct pattern was found for…

Woman in 30s With FAP Develops Kidney Disease Due to Tegsedi: Case

A woman with familial amyloid polyneuropathy (FAP) developed a type of kidney disease called focal segmental glomerulosclerosis — characterized by scarring in the kidneys — several months after starting treatment with Tegsedi (inotersen), according to a recent case report. Once the patient stopped taking Tegsedi, the kidney…

Amvuttra for FAP Among Specialty Treatments Offered by Soleo Health

Soleo Health, a U.S. national provider of pharmacy services, announced that Amvuttra (vutrisiran), an approved treatment for adults with familial amyloid polyneuropathy (FAP), has been added to its portfolio of specialty treatments. Amvuttra was developed by Alnylam Pharmaceuticals to treat FAP, also known as hereditary transthyretin-mediated (hATTR) amyloidosis with…