Disease-modifying medications can significantly improve survival outcomes among people with familial amyloid polyneuropathy (FAP) no matter what age the disease…
Articles by Marisa Wexler, MS
The U.S. Food and Drug Administration (FDA) has approved eplontersen for treating familial amyloid polyneuropathy (FAP). The therapy, which…
Screening for toxic protein clumps using biopsies and advanced imaging techniques may help people carrying mutations that cause hereditary transthyretin…
More than a year of treatment with the experimental therapy eplontersen significantly reduces blood levels of transthyretin — the…
Testing the ability to sense temperatures and mechanical forces could help predict when symptoms will appear for people who carry…
Treatment with Vyndaqel (tafamidis) reduces signs of heart damage for most people with hereditary ATTR amyloidosis with cardiomyopathy (hATTR-CM)…
Eplontersen continues to safely and effectively ease symptoms and improve quality of life for people with familial amyloid…
Treatment with Onpattro (patisiran) for 1.5 years leads to sustained reductions in disability progression among people with ATTR amyloidosis…
Treatment with Onpattro (patisiran) or Amvuttra (vutrisiran) leads to a reduction in blood levels of neurofilament light chain…
People with hereditary transthyretin amyloidosis (hATTR) who are treated with a combination of therapies appear to have better health outcomes…