Novo Nordisk is moving forward on a global Phase 2 clinical trial evaluating its experimental treatment NNC6019 in people with hereditary transthyretin amyloidosis (ATTR)…
News
Glu89Lys, a rare gene mutation that can cause familial amyloid polyneuropathy (FAP) — and is associated with early onset disease and a mix of…
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…
Alnylam Pharmaceuticals will not conduct an interim analysis of its Phase 3 clinical trial testing Amvuttra (vutrisiran) in people with a…
Familial amyloid polyneuropathy (FAP) patients who carry the Val30Met mutation may experience central nervous system involvement early in the disease course, likely before…
Older age and worse disease were significantly associated with cognitive impairments in people with familial amyloid polyneuropathy (FAP) who underwent a liver transplant, a…
A woman with familial amyloid polyneuropathy (FAP) developed a type of kidney disease called focal segmental glomerulosclerosis — characterized by scarring in the…
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…
Treatment with Tegsedi (inotersen) for more than three years continued to slow disease progression and improve the quality of life in people with…
The European Commission has approved Amvuttra (vutrisiran) as a treatment for familial amyloid polyneuropathy (FAP) — also known as hereditary transthyretin-mediated (hATTR) amyloidosis…
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