Article

A precision medicine approach for <i>HCN1</i> Developmental and Epileptic Encephalopathy

2024-01-11

Abstract excerpt

Pathogenic variants in HCN1 causing cation leak result in a severe developmental and epileptic encephalopathy (DEE). Current treatment options for patients with HCN1 -DEE are limited and are insufficient to fully address both the seizures and clinical comorbidities of this disorder. Org 34167 is a brain penetrant broad-spectrum HCN channel inhibitor that has completed phase I clinical trials. We used a range of...

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Literature Corpus work: 117993b9-71cc-550f-9246-a354f498d2c7
DOI: 10.1101/2024.01.09.574555

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