hATTR-PN

A newly developed second-generation transthyretin (TTR) protein stabilizer may be more effective at treating forms of TTR amyloidosis (ATTR), including familial amyloid polyneuropathy (FAP), than one already tested in patients, scientists in Spain announced. Called PITB, the molecule was seen to be better than tolcapone — a TTR…

My mother-in-law has always valued her autonomy. The pain of her peripheral neuropathy, the hallmark symptom of familial amyloid polyneuropathy (FAP), leads her to feel helpless at times. This helplessness gives way to grief and despair. Our family does our best to let our…

It has been 17 months since my husband, Aubrey, passed away due to complications of hereditary ATTR amyloidosis. Following his diagnosis in 2013, Aubrey’s journey was wrought with many untold challenges. His pain and struggle were visible to our family, including our four children. As his wife…

Attralus’ AT-01 may be more sensitive than a standard imaging agent at detecting toxic transthyretin (TTR) protein clumps in the heart of people with hereditary ATTR amyloidosis, a group of conditions that includes familial amyloid polyneuropathy (FAP), according to new data from a clinical trial. The findings suggest that…

More than a year of treatment with the experimental therapy eplontersen significantly reduces blood levels of transthyretin — the protein that accumulates to toxic levels in familial amyloid polyneuropathy (FAP) — eases nerve damage-related disability, and improves quality of life for patients. That’s according to the full results…

Testing the ability to sense temperatures and mechanical forces could help predict when symptoms will appear for people who carry mutations that cause familial amyloid polyneuropathy (FAP). That’s according to “Quantitative sensory testing and skin biopsy findings in late-onset ATTRv presymptomatic carriers: Relationships with predicted time of disease…

My mother-in-law, who has familial amyloid polyneuropathy (FAP), consistently adheres to her prescribed treatments, which has improved her mobility and overall mood. Because of this, she can handle more physical activity during family time. This positive change over the past few years, including those when I took a…

A committee of the U.S. Food and Drug Administration (FDA) has endorsed Onpattro (patisiran) for the treatment of the ATTR amyloidosis with cardiomyopathy (ATTR-CM), or heart damage. Earlier this year, Alnylam Pharmaceuticals, which is developing Onpattro, submitted a supplemental new drug application (sNDA) to the federal agency…

Medication adherence is crucial to managing familial amyloid polyneuropathy (FAP). Left untreated, FAP can have life-threatening consequences. As an article published in U.S. Pharmacist notes, according to the World Health Organization (WHO), medication adherence can have a more direct impact on patient outcomes than even the…

An international panel of patient advocates and healthcare providers has published the first set of recommendations outlining steps for the holistic care of people living with hereditary transthyretin-mediated amyloidosis (hATTR), a group of disorders that includes familial amyloid polyneuropathy (FAP). The guidelines focus not only on early diagnosis…