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Blog Archive

Monday, August 11, 2014

RE(ACT) TMAU project submitted by Dr John Christodoulou

Dr John Christodoulou TMAU project
Dr John Christodoulou
REA(CT) TMAU project has been submitted

We can inform you that a TMAU project has be submitted to the RE(ACT) rare disease crowdfunding platform :

by Dr John Christodoulou et al of Sydney Australia
It will now be reviewed by the RE(ACT) Scientific committee (expected time : 3-5 weeks)
NOTE : As it is exploratory it may not be approved by the committee

Project :
It has 2 parts :
1. is to use Ataluren to see if it improves nonsense mutation TMAU cases. This is a severe type of mutation and most sufferers probably do not have nonsense mutations (about 10% are estimated to have nonsense mutations).
TMAU project
2. To see if a microbe 'methylophilus methotropholus' could be used as a specific 'probiotic' for TMAU patients as it may neutralize trimethylamine in the gut. This should in theory helkp all TMAU sufferers.

Time and Cost :
The study would be expected to take 3 years, with 2 professionals being employed especially to conduct the study.
The cost would be around $AUS 473,000 plus RE(ACT) admin fees of 10% (current estimate)
The study would presumably take place in Australia

We will elaborate more over the days and weeks on the project, but basically all the info of the project is in the photo on the right, which is a copy of the document submitted.

To the right is Professor Christodoulou's career summary

Click to read project in full (pdf hosted on MEBO website)
Dr Christodoulou TMAU project  

Dr Christodoulou career summary 
(pdf hosted on MEBO website)

Other links :
RE(ACT) TMAU Group
Dr Christodoulou researcher listed on RE(ACT)

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