The U.S. Food and Drug Administration (FDA) has granted orphan drug status to ART001, a gene editing candidate that Accuredit Therapeutics is developing as a one-time treatment for transthyretin (ATTR) amyloidosis, a group of diseases that includes familial amyloid polyneuropathy (FAP). Such a designation supports developing possible treatment…
The U.S. Food and Drug Administration (FDA) has extended the approval of Amvuttra (vutrisiran) to adults with cardiomyopathy (heart damage) due to nonhereditary or hereditary transthyretin amyloidosis (ATTR-CM). Alnylam Pharmaceuticals’ Amvuttra was specifically cleared for use to reduce death, hospital stays, and urgent visits due to heart problems in…
Wainzua (eplontersen) is now approved in the European Union (EU) for treating adults with early-stage familial amyloid polyneuropathy (FAP). With the European Commission’s approval, Wainzua can treat adults in the first two stages of FAP — stage 1, when abnormal sensations appear, but the patient is still able to…
Alnylam Pharmaceuticals is planning to launch a Phase 3 clinical trial toward the end of this year to test its therapy candidate nucresiran in people with familial amyloid polyneuropathy (FAP) — though details on the trial’s design are still being discussed with global regulators. That’s according to pipeline…
Throughout March — Amyloidosis Awareness Month — the Amyloidosis Foundation is inviting the public to join its “Light the Night for Amyloidosis” campaign, increasing awareness about a group of rare diseases that includes familial amyloid polyneuropathy (FAP). People are asked to brighten the entrance or features of their…
Examining small samples of tissue from the skin and the salivary glands, those that produce saliva, increases the chances of detecting the toxic protein clumps that cause familial amyloid polyneuropathy (FAP) early in the disease’s course, a study reported. Its findings suggest that analyzing both these tissues, whose collection…
Five years of treatment with Onpattro (patisiran) slowed disability progression and maintained quality of life for people with familial amyloid polyneuropathy (FAP) in a long-term clinical trial, a study shows. Although Onpattro can help delay FAP progression, “patients tend not to recover function that is lost before starting…
People with familial amyloid polyneuropathy (FAP) who were given disease-modifying treatments in routine clinical care show relatively stable disease over time, but a substantial proportion still showed signs of disease progression throughout follow-up, a real-world study in France shows. “In routine care, the overall population of patients remains stable,”…
Researchers have uncovered previously unknown structural features of transthyretin, the protein that’s faulty in familial amyloid polyneuropathy (FAP), which could pave the way toward new treatment strategies. “We’ve unveiled a molecular complexity that has been hidden from researchers for decades, which enables us to design better medicines to stabilize…
People with familial amyloid polyneuropathy (FAP) have fewer autonomic symptoms caused by damage to the nerves that control involuntary bodily functions after more than 1.5 years of treatment with Wainua (eplontersen) compared with a placebo. That’s according to a new secondary analysis from the Phase 3 NEURO-TTRansform…