A rare mutation in the TTR gene — mutations in which are the cause of hereditary transthyretin amyloidosis (hATTR) —…
Articles by Margarida Maia, PhD
Six months of treatment with Amvuttra (vutrisiran) appears to maintain or slightly improve clinical status and quality of life…
Blood levels of the transthyretin (TTR) protein at the start of Vyndaqel (tafamidis) treatment and after one month may help…
Attruby (acoramidis) slows disease progression and significantly reduces death and cardiovascular-related hospitalizations in adults with hereditary or wild-type…
People with transthyretin amyloid cardiomyopathy (ATTR-CM) live longer when the disease is diagnosed and treated earlier, with treatment benefits…
The number of people diagnosed with transthyretin amyloidosis (ATTR) in the U.S. is climbing, a surge researchers attribute to…
Adults who carry the Val30Met, or V30M, mutation — the most common cause of early-onset hereditary transthyretin amyloidosis with…
Polyneuropathy, or widespread nerve damage, occurs in both hereditary and wild-type transthyretin amyloidosis (ATTR), but the hereditary form, which includes…
Onpattro (patisiran) safely and effectively helps adults with familial amyloid polyneuropathy (FAP) caused by a V122I or T60A…
Alnylam Pharmaceuticals has launched a Phase 3 clinical trial testing its experimental treatment nucresiran in adults with familial…