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Ataluren

Question
Hello,

My son has a mutation concerned by Ataluren, I would like to know if new results have been publish regarding Ataluren development?
Thank you in advance.
Answer
Hello,
Here are the last results available regarding Ataluren development:
“For the international phase III trial, 238 patients over 6 years with a stop mutation were included. At random, patients received for 48 weeks, the ataluren or the placebo.
The results showed that patients with ataluren had lower decline in lung function and less pulmonary exacerbations, compared to those who took a placebo. But this difference was not significant.
It seems that inhaled antibiotics have a negative effect on the action of ataluren. When analyzing only the data of patients not treated chronically by inhaled antibiotics, the decline in function and number of pulmonary exacerbations is lower for those who took the ataluren compared with placebo. This time the difference between the two groups is greater and therefore significant.”

We hope that the pharmaceutical company, PTC Therapeutics, which develops ataluren, communicates on the next steps for this molecule.


Best regards
Anna Ronayette
28.02.2013