Treatment switches in people with familial amyloid polyneuropathy (FAP) are common, with Amvuttra (vutrisiran) emerging as a preferred option due to its more…
News
A Phase 3 clinical trial that’s testing the oral treatment acoramidis to prevent or delay disease onset is continuing to recruit adults who carry a…
Amvuttra (vutrisiran) appears to be safe for long-term use in adults with familial amyloid polyneuropathy (FAP) and those with another form of transthyretin…
HATTR-PN
NewsGene therapy nex-z lowers FAP protein levels for 3 years
A single dose of gene-editing therapy nexiguran ziclumeran (nex-z) leads to deep reductions in blood levels of the disease-causing transthyretin (TTR) protein in adults with…
Yoltech Therapeutics has raised about $45 million in funding to advance its clinical programs, including YOLT-201, an experimental gene-editing therapy for familial amyloid…
There is a high prevalence of the Val30Met (V30M) TTR gene mutation — the most common mutation associated with familial amyloid polyneuropathy (FAP) and…
Hereditary transthyretin amyloidosis (ATTRv), a group of diseases that includes familial amyloid polyneuropathy (FAP), might not be as easily categorized into distinct neurological and…
Specialists in Scandinavia have updated the Kumamoto scale, a tool long used to monitor the progression of hereditary transthyretin amyloidosis (ATTRv), a group of diseases…
HATTR-PN
NewsEarly-stage FAP brings heavy economic burden for society
People with early-stage familial amyloid polyneuropathy (FAP) cost society more than twice as much as symptom-free carriers of disease-causing genetic mutations, largely due to…
Early genetic screening for people with unexplained nerve damage, or neuropathy, may help uncover undiagnosed cases of familial amyloid polyneuropathy (FAP), allowing faster access…
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