This is fantastic news. Orphan Drug Status - upwards and on-wards towards a marketable product called Trofinetide (nickname Trof). Fragile X, Concussion and Autism treatment to follow. Again the ultimate aim, business wise, is to store Trof at sports grounds and in ambulances around the globe!
But also just as important -probably more - to offer a treatment for so many afflictions that currently have next to no treatment.
FDA Grants Orphan Drug Designation, page-20
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