[Review of the recent literature on peripheral neuropathies: Therapeutic advances.]
Le Kremlin-Bicêtre, France. In Rev Neurol (paris), 18 Dec 2013
In familial amyloidotic polyneuropathy, a multicentric controlled study against placebo with tafamidis, an akinetic stabilizer of transthyretin (TTR) 20mg/d, in early stage of Val30MetTTR showed efficiency in the evaluable group and led to marketing authorization by the EMA in stage 1 to slow the progression of the neuropathy.
[What's new in hereditary neuropathies ?]
Lille, France. In Rev Neurol (paris), 11 Dec 2013
UNLABELLED: This short review highlights five studies published in 2012 in the field of Charcot-Marie-Tooth disease (CMT) and transthyretin familial amyloid neuropathies (TTR-FAN).
[Familial amyloid polyneuropathies: therapeutic issues].
Le Kremlin-Bicêtre, France. In Bull Acad Natl Med, Oct 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, Oct 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, Mar 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.
Familial amyloid polyneuropathy.
Créteil, France. In Lancet Neurol, 2011
Nerve lesions are induced by deposits of amyloid fibrils, most commonly due to mutated transthyretin (TTR).