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an FDA advisory committee rejected adding it to the approved compounding list in 2024, citing toxicity and an unresolved cardiac signal

These advances in disease mechanism research provide the basis for the design of drugs such as rapamycin (mTOR) inhibitors (tuberous sclerosis 22 and fragile X syndrome, 23,24 ) metabolic glutamate receptor (mGluR) antagonists (fragile X syndrome 25 and 16p11.2 deletion 26 ), and insulin growth factor (IGF-1) (Rett syndrome 27 and PhelanMcDermid syndrome 28,29 )

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