Dr John Christodoulou |
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 |
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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