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Beyond Seizures as an Outcome Measure: A Global Severity Scoring System for CDKL5 Deficiency Disorder

CDKL5 deficiency disorder (CDD) is a rare developmental and epileptic encephalopathy (DEE) associated with multiple impairments and comorbidities. Outcome measures for disease-modifying clinical trials for DEEs should measurably capture a spectrum of caregiver priorities and be externally validated.

Citation:
Jacoby P, Marsh ED, Demarest S, Saldaris JM, Leonard H, Olson HE, ……. Downs J, Benke TA. Beyond Seizures as an Outcome Measure: A Global Severity Scoring System for CDKL5 Deficiency Disorder. Brain and Behavior. 2025;15(11).

Keywords:
CDKL5 deficiency disorder; biomarkers; epilepsy; healthcare; outcome assessment; quality of life

Abstract:
CDKL5 deficiency disorder (CDD) is a rare developmental and epileptic encephalopathy (DEE) associated with multiple impairments and comorbidities. Outcome measures for disease-modifying clinical trials for DEEs should measurably capture a spectrum of caregiver priorities and be externally validated.