England’s National Institute for Health and Care Excellence (NICE) has issued a draft guidance recommending Amvuttra (vutrisiran) be available…
Articles by Marisa Wexler, MS
Eye involvement can become common over time in people with hereditary transthyretin amyloidosis (hATTR), a group of disorders that includes…
Alnylam Pharmaceuticals has submitted an application to the U.S. Food and Drug Administration (FDA) asking the agency to approve…
One-time treatment with the experimental gene-editing therapy NTLA-2001 led to substantial reductions in the levels of toxic transthyretin protein that…
A woman with familial amyloid polyneuropathy (FAP) developed a type of kidney disease called focal segmental glomerulosclerosis —…
The European Commission has approved Amvuttra (vutrisiran) as a treatment for familial amyloid polyneuropathy (FAP) — also known…
Onpattro (patisiran), a widely approved treatment for familial amyloid polyneuropathy (FAP), preserved functional ability and improved life quality…
Alnylam Pharmaceuticals has selected Orsini Specialty Pharmacy to distribute Amvuttra (vutrisiran), which was recently approved by the U.S.
A one-time treatment with NTLA-2001, Intellia Therapeutics’ experimental gene-editing therapy for people with familial amyloid polyneuropathy (FAP) and other…
Several cases in which treatment with Onpattro (patisiran) was used to ease familial amyloid polyneuropathy (FAP) symptoms were…