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A Prospective Natural History Study Protocol for Clinical Trial Readiness in Synaptic Disorders

2026-02-02

Abstract excerpt

<h4>Structured Abstract</h4> <h4>Objective</h4> STXBP1- Related Disorders ( STXBP1 -RD) and SYNGAP1 -Related Disorder ( SYNGAP1 -RD) are two common genetic synaptopathies, leading to epilepsy, developmental delay, and intellectual disability. Both STXBP1 -RD and SYNGAP1 -RD are potential targets for disease-modifying therapies, but there is limited information in the literature describing the natural histor...

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Literature Corpus work
583265a6-ee68-578c-9618-a2b6a4176346
DOI
10.64898/2026.01.30.26344887
Open publication

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A Prospective Natural History Study Protocol for Clinical Trial Readiness in Synaptic DisordersDOI 10.64898/2026.01.30.26344887
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