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MIGLUSTAT
- Question
- Hello,
The latest results of Miglustat's clinical trials were not those expected. Are researches on this molecule definitively abandoned? Thank you in advance for your response
- Answer
- Hello,
Two phase II trials, conducted by the pharmaceutical company Actelion (Headquarters: Switzerland) took place first in Spain then in Belgium. Indeed the results of these trials have failed to draw clear conclusions about the ability of Miglustat to restore the function of the CFTR protein in patients with the F508del mutation. The data showed considerable variability between patient treated by the same treatment (miglustat or placebo).
However, research does not stop and a new Phase II trial to be conducted only in France will soon emerge.
Best regards
Anna Ronayette
- 30.07.2012
