Glu89Lys, a rare gene mutation that can cause familial amyloid polyneuropathy (FAP) — and is associated with early onset disease and a mix of cardiac, neurological, and eye problems — was found to be endemic in Spain, a recent study reported. Researchers conducted a DNA analysis of seven families…
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…
Alnylam Pharmaceuticals will not conduct an interim analysis of its Phase 3 clinical trial testing Amvuttra (vutrisiran) in people with a form of heart disease called transthyretin-mediated (ATTR) amyloidosis with cardiomyopathy. Instead, the company will wait for the initial findings of final analyses of key safety…
Familial amyloid polyneuropathy (FAP) patients who carry the Val30Met mutation may experience central nervous system involvement early in the disease course, likely before symptoms are noticeable, according to a small study. Involvement of the central nervous system (CNS, the brain and spinal cord) follows a three-stage progression, its researchers…
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…
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…
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…
Treatment with Tegsedi (inotersen) for more than three years continued to slow disease progression and improve the quality of life in people with familial amyloid polyneuropathy (FAP), according to new data from the extension study of the NEURO-TTR trial. Patients who started treatment earlier experienced greater benefits, but…
The European Commission has approved Amvuttra (vutrisiran) as a treatment for familial amyloid polyneuropathy (FAP) — also known as hereditary transthyretin-mediated (hATTR) amyloidosis with polyneuropathy — for people in the EU with stage 1 or stage 2 polyneuropathy. The decision was announced by the medication’s developer, Alnylam…
Onpattro (patisiran), a widely approved treatment for familial amyloid polyneuropathy (FAP), preserved functional ability and improved life quality for people with a related form of heart disease called transthyretin-mediated (ATTR) amyloidosis with cardiomyopathy. That’s according to top-line results from the Phase 3 APOLLO-B clinical trial, which tested the…