These results raise fascinating questions that we may not know the answer to for a long time.
How much can the drug improve a patient?
Does improvement keep occurring indefinitely?
What is most intriguing is can the drug prevent the onset and development of the neurological affect altogether if treatment commenced at its onset (6-18 months of age)?
Maybe screening for deficiencies in MECP2 should be routine in infancy?
So in other words do we have a treatment or a cure, and if it is “just” a treatment can it mimic a cure if treatment is initiated early enough?
Ann: Positive topline results from Phase 3 trial in Rett syndrome, page-47
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