Observational studyNeurology2025
Natural History of Children and Adolescents With Dravet Syndrome: A 24-Month Follow-Up.
Observational study in Neurology, 2025. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Cited by 4 papers.
What it found
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The abstract states no effect estimate the extractor could read, or names no intervention and outcome on the map, so this paper lights no cell and moves no belief. It is still indexed, cited and linked below.
The trial behind it
Trials whose registry record cites this paper, or whose number appears in the abstract. A trial that started after this paper was published is citing it as background, not reporting it.
Neither the registry nor the abstract names a trial number. If this is a trial report, that itself is worth knowing.
Who cites it
4 citing papers in PubMed.
- Cognitive and behavioral clinical outcome assessments in children with developmental and epileptic encephalopathies: Issues and instruments.Epilepsia · 2026Review
- Ameliorating Seizures in Dravet Syndrome: A Review of Newly Approved and Investigational Drugs, RNA and Gene-Based Therapies.CNS drugs · 2026Review
- CharacterizingmedRxiv : the preprint server for health sciences · 2026Article
- Article
Corrections and comments
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Authors and funding
15 authors.
Funding
No grant is acknowledged in the PubMed record.
Abstract
BACKGROUND AND
objectivesDravet syndrome (DS) is an intractable developmental and epileptic encephalopathy caused primarily by pathogenic variants in the voltage-gated sodium channel α subunit 1 (
methodsBUTTERFLY was a US-based multicenter, longitudinal observational study involving patients with genetically confirmed DS who were receiving standard-of-care treatment. Patients aged 2-18 years with a confirmed DS diagnosis were included. Exclusion criteria included gain-of-function
resultsOf the 36 patients enrolled, 21 completed the study. At baseline, the mean (SD) patient age was 10.8 (5.2) years, and 61.1% were female. Disease progression modeling indicated no significant improvements in 4 of the 6 Vineland-3 subdomains analyzed over 24 months. Receptive Communication (+7.24; 95% CI 1.30-13.19; DISCUSSION: Despite a relatively small sample size and high withdrawal rate, BUTTERFLY data reveal a widening developmental gap between patients with DS and children with typical development over 24 months. These findings reinforce the critical need for disease-modifying therapies that address the underlying genetic cause of DS to improve long-term outcomes.
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