"It is essential to conduct longer term studies in adults as well as conduct similar studies in younger individuals."
That doesn't mean necessarily that it's the usual route. The fda has been known to approve drugs for use in similar circumstances and the further studies are conducted on some of those users.
Retts is a life threatening illness with no treatment whatsoever, and that makes it a very special case.
We will know early in 2015 what will happen.
As regarding funding. neu seems to be able to run on the smell of an oily rag, and any further study requirements will attract much funding from action groups and governmental bodies.
Rett, page-5
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