Perhaps:
https://www.fda.gov/ForPatients/Other/ExpandedAccess/ucm20041768.htm
"Expanded access, also called “compassionate use,” provides a pathway for patients to gain access to investigational drugs, biologics and medical devices for serious diseases or conditions"
Fragile X passes the test for "serious disease" and potentially Rett Syndrome does too. The quality of life is terrible for the patients of both and they also experience reduced life expectancy.
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