missfizi
i just got this on a tmau yahoo group, if this is for real, i would be totally happy.
"Ataluren (PTC124) finishes it's Phase III trial for Cystic Fybrosis on July 9 - If it passes this, the FDA could approve it in a couple of months and it could be commercially available for us to experiment with.
The only side effects reported so far have been Nausea in patients given very high dosages - the other side effect which was reported with normal dosage was an increase in the production of certain liver enzymes which was seen as a harmless side effect. As our livers don't produce enough of the FMO3 enzyme, this gives me great hope.
Ataluren is the drug recommended by Dr John Christodoulou to be trialed in the TMAU research proposal, which wouldn't exist if not for the great work Rob Brown has done for all of us. The big problem here is funding - if Ataluren gets released I have personally decided to try it for myself - no one knows if it will work on us - but the
Science says we have a good chance"