PMID- 25858436
OWN - NLM
STAT- MEDLINE
DCOM- 20160216
LR  - 20150527
IS  - 1532-2777 (Electronic)
IS  - 0306-9877 (Linking)
VI  - 85
IP  - 1
DP  - 2015 Jul
TI  - Is riluzole a potential therapy for Rett syndrome?
PG  - 76-8
LID - S0306-9877(15)00140-1 [pii]
LID - 10.1016/j.mehy.2015.03.025 [doi]
AB  - Rett syndrome (RTT) is a severe neurodevelopmental disorder with autistic 
      features and is caused by loss-of-function mutations in the gene encoding 
      methyl-CpG-binding protein 2 (MECP2) in the majority of cases. Besides 
      symptomatic treatment, no therapeutic trials have shown effectiveness for RTT. 
      Some perspectives in the treatment of RTT have been provided by recent works 
      showing a phenotypic reversal by increasing brain-derived neurotrophic factor 
      (BDNF) expression in a RTT mouse model. Glutamate may also play an important role 
      in the primary pathogenesis in Rett syndrome through the excitotoxic neuronal 
      injury in experimental models. Riluzole, an agent currently approved for the 
      treatment of amyotrophic lateral sclerosis, is a glutamatergic modulator and BDNF 
      enhancer with neuroprotective properties. For these reasons, riluzole could 
      potentially play an important role in the treatment of RTT symptoms. Several 
      points regarding the use of riluzole in RTT are discussed. Further evaluation of 
      the therapeutic effects of this agent in RTT animal models is needed before 
      clinical trials can begin.
CI  - Copyright (c) 2015 Elsevier Ltd. All rights reserved.
FAU - Tsai, Shih-Jen
AU  - Tsai SJ
AD  - Department of Psychiatry, Taipei Veterans General Hospital, Taiwan; Division of 
      Psychiatry, School of Medicine, National Yang-Ming University, Taiwan. Electronic 
      address: tsai610913@gmail.com.
LA  - eng
PT  - Journal Article
DEP - 20150404
PL  - United States
TA  - Med Hypotheses
JT  - Medical hypotheses
JID - 7505668
RN  - 0 (Neuroprotective Agents)
RN  - 7LJ087RS6F (Riluzole)
SB  - IM
MH  - Humans
MH  - Models, Theoretical
MH  - Neuroprotective Agents/*therapeutic use
MH  - Rett Syndrome/*drug therapy
MH  - Riluzole/*therapeutic use
EDAT- 2015/04/11 06:00
MHDA- 2016/02/18 06:00
CRDT- 2015/04/11 06:00
PHST- 2015/02/09 00:00 [received]
PHST- 2015/03/14 00:00 [revised]
PHST- 2015/03/28 00:00 [accepted]
PHST- 2015/04/11 06:00 [entrez]
PHST- 2015/04/11 06:00 [pubmed]
PHST- 2016/02/18 06:00 [medline]
AID - S0306-9877(15)00140-1 [pii]
AID - 10.1016/j.mehy.2015.03.025 [doi]
PST - ppublish
SO  - Med Hypotheses. 2015 Jul;85(1):76-8. doi: 10.1016/j.mehy.2015.03.025. Epub 2015 
      Apr 4.