Thanks. Your comment about quality-of-life improvement is a valid one, given that the impeccable safety record is maintained in larger studies (I don't see why it wouldn't). I would be interested to know what the economics might look like, given OD designation is already awarded to Daybue in the US and EU which gives 7- and 10-year marketing exclusivity. I suppose it can potentially be used as a combination therapy, but I don't know how that would look from a pricing and reimbursement perspective
Ann: Positive Top-Line Rett Syndrome Clinical Trial Results, page-21
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