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results regarding Ataluren project
- Question
- Hello,
I am the father of a one year old CF girl with a stop codon mutation and I received by mail the first results of phase 3 Ataluren (PTC124).
All information that I received are in english, can you enlighten me on these results?
- Answer
- Hello,
For this international phase III trial, 238 patients over 6 years with a stop mutation were included. At random, patients received for 48 weeks, 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, will communicate on the next steps for this molecule.
Best regards
Anna Ronayette - 23.07.2012
