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STXBP1 Encephalopathy with Epilepsy
Clinical trial pipeline · Data from ClinicalTrials.gov
See which STXBP1 Encephalopathy with Epilepsy trials you may qualify forClinical trial pipeline · Data from ClinicalTrials.gov
See which STXBP1 Encephalopathy with Epilepsy trials you may qualify forSTXBP1-related disorders (STXBP1-RD) are rare genetic neurodevelopmental disorders, caused by pathogenic variants in the gene STXBP1. The core clinical features…
The purpose of this study is to find out more about STXBP1 and SYNGAP1 related disorders. The information gathered by this study will be used to prepare for cli…
Simons Searchlight is an observational, online, international research program for families with rare genetic variants that cause neurodevelopmental disorders a…
The aim of this pilot study is to explore whether the knowledge and experience gained during the T-GaiD project (Treatment of Gait Disorders in Dravet Syndrome…
This study is to evaluate the use of glycerol phenylbutyrate for monogenetic developmental epileptic encephalopathies (DEEs). DEEs are characterized by epilepsy…
The goal of this clinical trial is to learn about the safety of CAP-002 gene therapy in children with Syntaxin-Binding Protein 1 (STXBP1) Encephalopathy. It wil…