Main reason for sharing information was to emphasise rarity of Rett syndrome with 60 patients and positive result Neuren may be in good position to start negotiation about filling soon after the result (if positive) , by the time filling happens 12-18 months extension study data will be available
rarity of disease and non availability of treatment is both considered when accepting drugs with limited clinical trial data, fragile X syndrome data will be available in 2015 and post traumatic brain injury will certainly need phase III trials
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