Paper Information

Journal:   GENETICS IN THE 3RD MILLENNIUM   WINTER 2016 , Volume 14 , Number 1; Page(s) 0 To 0.
 
Paper: 

CLINICAL TRIALS AND BIOMARKERS IN CMT

 
 
Author(s):  ATTARIAN SHAHRAM*
 
* REFERENCE CENTER FOR NEUROMUSCULAR DISORDERS AND ALS TIMONE HOSPITAL UNIVERSITY, MARSEILLE, FRANCE
 
Abstract: 

Charcot-Marie-Tooth (CMT) diseases represent a heterogeneous genetic disorder with more than 80 genes implicated, however sharing a similar phenotype. In recent years, advances in molecular genetics and molecular biology, and also the development of various animal models of CMT, have led to a better understanding. This knowledge represents a prerequisite for the development of future therapies in CMT.
In this review, we discuss current research progress and recent promising preclinical work for CMT1A and the development of new biomarkers in CMT. We also describe CMT therapeutic development and ongoing clinical trials. While no effective therapy is known to date, ongoing new therapeutic trials such as PXT3003 (a low dose combination of the three already approved drugs baclofen, naltrexone, and D-sorbitol) give hopes for potential curative treatment.
This summary of CMT research identifies some of the gaps which may have an impact on upcoming clinical trials.

 
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