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…
hATTR-PN
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,”…
In my last column, I mentioned that my mother-in-law was recently diagnosed with rheumatoid arthritis. She already suffers from the symptoms of familial amyloid polyneuropathy (FAP), including itching and burning sensations in her hands and feet. The pain of her neuropathy symptoms makes it hard for her to walk…
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…
For neuropathy patients and caregivers, having a purpose in life may go a long way toward helping us manage peripheral neuropathy. Many familial amyloid polyneuropathy (FAP) patients and caregivers, however, are forced to put a great deal of focus on simply surviving. My mother-in-law has tingling and burning…
It’s another stunning summer day here in New Zealand’s Waikato region. We’ve been blessed all week with clear skies and a warm breeze blowing through the house, as the blinds are drawn up and the backyard doors are open. View of the Waikato region in New Zealand. (Photo by Jaime…
A single infusion of YOLT-201, Yoltech Therapeutics’ investigational gene-editing therapy, appears to be safe and to effectively reduce blood levels of the disease-driving TTR protein in people with familial amyloid polyneuropathy (FAP). That’s according to preliminary data from six FAP patients given the therapy in the initial,…
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- The emotional burden of receiving negative genetic test results