PMID- 25416603
OWN - NLM
STAT- MEDLINE
DCOM- 20150901
LR  - 20141127
IS  - 1744-8360 (Electronic)
IS  - 1473-7175 (Linking)
VI  - 14
IP  - 12
DP  - 2014 Dec
TI  - Current and future treatment of amyloid neuropathies.
PG  - 1437-51
LID - 10.1586/14737175.2014.983905 [doi]
AB  - Amyloid neuropathies of acquired or genetic origin are disabling and 
      life-threatening, until recently there were few treatment options available. Poor 
      prognosis is related to progressive neuropathy and associated, although often 
      underdiagnosed, cardiac involvement in specific transthyretin (TTR) gene 
      mutations. Recent progress has modified prognosis and management of amyloid 
      neuropathies. In TTR-familial amyloidosis with polyneuropathy, major changes have 
      occurred over the last 30 years: better knowledge concerning genetics, phenotypes 
      and epidemiology, and the advent of possible treatments. Liver transplantation, 
      first performed in 1990, stopped disease progression, thus doubling survival in 
      early onset V30M patients. More recently tetramer stabilizers (Tafamidis and 
      Diflunisal) showed a significant reduction of progression of neuropathic scores; 
      Tafamidis is now recommended in Stage I patients. Two multicentric clinical 
      trials are now ongoing to evaluate TTR gene silencing by antisense 
      Oligonucleotides (ASO) or siRNA. In the near future we should have new 
      therapeutical options for patients with amyloid neuropathy.
FAU - Adams, David
AU  - Adams D
AD  - Department of Neurology, CHU Bicetre, APHP, Le Kremlin Bicetre, Cedex, France.
FAU - Cauquil, Cecile
AU  - Cauquil C
FAU - Theaudin, Marie
AU  - Theaudin M
FAU - Rousseau, Antoine
AU  - Rousseau A
FAU - Algalarrondo, Vincent
AU  - Algalarrondo V
FAU - Slama, Michel S
AU  - Slama MS
LA  - eng
PT  - Journal Article
PT  - Review
DEP - 20141121
PL  - England
TA  - Expert Rev Neurother
JT  - Expert review of neurotherapeutics
JID - 101129944
RN  - 0 (Benzoxazoles)
RN  - 0 (Prealbumin)
RN  - 8FG9H9D31J (tafamidis)
SB  - IM
MH  - Amyloid Neuropathies/genetics/*therapy
MH  - Animals
MH  - Benzoxazoles/therapeutic use
MH  - *Genetic Therapy
MH  - Humans
MH  - *Liver Transplantation
MH  - Mutation/*genetics
MH  - Prealbumin/*genetics
OTO - NOTNLM
OT  - TTR gene silencing
OT  - TTR tetramer stabilizer
OT  - antisense oligonucleotides
OT  - diflunisal
OT  - familial amyloid polyneuropathy
OT  - liver transplantation
OT  - patisiran
OT  - tafamidis
OT  - therapeutic patient education program
OT  - transthyretin
EDAT- 2014/11/25 06:00
MHDA- 2015/09/02 06:00
CRDT- 2014/11/23 06:00
PHST- 2014/11/23 06:00 [entrez]
PHST- 2014/11/25 06:00 [pubmed]
PHST- 2015/09/02 06:00 [medline]
AID - 10.1586/14737175.2014.983905 [doi]
PST - ppublish
SO  - Expert Rev Neurother. 2014 Dec;14(12):1437-51. doi: 10.1586/14737175.2014.983905. 
      Epub 2014 Nov 21.