[Familial amyloid polyneuropathies: therapeutic issues].
Le Kremlin-Bicêtre, France. In Bull Acad Natl Med, 2012
The first-line specific treatment of choice for met30 TTR-FAP is liver transplantation (LT) which suppresses the main source of mutant TTR, halts the progression of neuropathy in 70% of cases, and doubles the median survival time.
[Familial amyloidotic polyneuropathies].
Limoges, France. In Bull Acad Natl Med, 2012
Transthyretin familial amyloid polyneuropathy (TTRFAP) is an autosomal dominant neuropathy that is fatal within about 10 years after symptom onset.
In Nat Rev Drug Discov, 2012
In November 2011, tafamidis (Vyndaqel; Pfizer), a small molecule that inhibits the dissociation of transthyretin tetramers, was granted marketing authorization by the European Commission for the treatment of transthyretin amyloidosis in adult patients with stage 1 symptomatic polyneuropathy to delay peripheral neurological impairment.