Just to put the wider market for off-label use in context, there are about 1.5 million kids in the US who are autistic, compared to 15,000 who have Rett Syndrome. An example of the off-label use of a prescription drug is Botox being used for appearance medicine, when its FDA approval was for the treatment of migraine. If this drug, or any of the others in the Neuren stable, proves effective in the treatment of autism spectrum disorders, the worldwide potential is just plain astronomical.
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