Two Italian brothers carrying V122I, a TTR gene mutation often linked to hereditary ATTR amyloidosis with cardiomyopathy (hATTR-CM), developed neurological symptoms that resembled familial…
hATTR-PN
HATTR-PN
ColumnsHow caregivers can play a role in interpreting medical information
The past few weeks have been challenging for my extended family and me. My mother-in-law, who has familial amyloid polyneuropathy (FAP), experienced a health…
A single infusion of NTLA-2001, an experimental gene-editing therapy, generally is safe and leads to a marked reduction in blood levels of the harmful transthyretin…
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),…
My mother-in-law has always valued her autonomy. The pain of her peripheral neuropathy, the hallmark symptom of familial amyloid polyneuropathy (FAP), leads…
HATTR-PN
ColumnsWhy communication is important for amyloidosis patients, caregivers
It has been 17 months since my husband, Aubrey, passed away due to complications of hereditary ATTR amyloidosis. Following his diagnosis in…
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…
More than a year of treatment with the experimental therapy eplontersen significantly reduces blood levels of transthyretin — the protein that accumulates to toxic…
HATTR-PN
NewsDetecting touch, temperature may help predict FAP onset
Testing the ability to sense temperatures and mechanical forces could help predict when symptoms will appear for people who carry mutations that cause familial…
HATTR-PN
ColumnsSetting goals may help patients get past their barriers to treatment
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…
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