A single dose of gene-editing therapy nexiguran ziclumeran (nex-z) leads to deep reductions in blood levels of the disease-causing transthyretin…
Articles by Lindsey Shapiro, PhD
There is a high prevalence of the Val30Met (V30M) TTR gene mutation — the most common mutation associated with…
Hereditary transthyretin amyloidosis (ATTRv), a group of diseases that includes familial amyloid polyneuropathy (FAP), might not be as easily…
Kidney disease is an underrecognized complication of hereditary transthyretin amyloidosis (ATTRv) — a group of diseases that includes familial…
People with familial amyloid polyneuropathy (FAP) who were given disease-modifying treatments in routine clinical care show relatively stable disease…
Adults with familial amyloid polyneuropathy (FAP) living in Canada may soon be able to receive Amvuttra (vutisiran) at…
Onpattro (patisiran) may help familial amyloid polyneuropathy (FAP) patients who respond poorly to tafamidis meglumine stabilize or…
Wainua (eplontersen) is able to slow disease progression and improve life quality for people with familial amyloid polyneuropathy…
The electrical signaling patterns associated with polyneuropathy, or damage to nerves outside the brain and spinal cord, vary in a…
A single infusion of NTLA-2001, an experimental gene-editing therapy, generally is safe and leads to a marked reduction in blood…