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They have been extensively studied in large-scale human clinical trials, with robust safety data spanning over a decade

Syndromic generally refers to mutations in a specific gene or genome, manidesting as neurological syndromes (such as fragile X syndrome, tuberous sclerosis, Rett syndrome, PhelanMcDermid syndrome and Angelman syndrome)

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For laboratory use, it may be reconstituted with bacteriostatic water or another suitable solvent according to research protocols