Natural History of Children and Adolescents With Dravet Syndrome
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.
Authors
- Pam Ventola
- J. Robert Flamini
- Kelly G. Knupp (ORCID: https://orcid.org/0000-0002-1967-0827)
- Tanmay S Chavan
- Kimberly A. Parkerson
- Julie Ziobro (ORCID: https://orcid.org/0000-0002-7250-7308)
- Barry Ticho
- Michael A. Ciliberto (ORCID: https://orcid.org/0000-0001-8188-7261)
- Miganush Stepanians
- Elaine C. Wirrell (ORCID: https://orcid.org/0000-0003-3015-8282)
- Dillon Y. Chen (ORCID: https://orcid.org/0000-0003-2270-1652)
- Muhammad Zafar (ORCID: https://orcid.org/0000-0003-2002-3907)
- Joseph Sullivan (ORCID: https://orcid.org/0000-0003-2081-8988)
- Nicole LaVallee
- Wang Fei (ORCID: https://orcid.org/0000-0003-1545-6919)
Institutions
- Linde (United States) (US)
- University of Iowa (US)
- Duke University (US)
- University of California, San Francisco (US)
- University of Michigan (US)
- Chengdu Research Base of Giant Panda Breeding (CN)
- Yale University (US)
- University of California San Diego (US)
- University of New Haven (US)
- Mayo Clinic in Arizona (US)
- PROMETRA International (SN)
- University of Colorado Denver (US)
Publication Details
- Journal
- Neurology
- Published
- 2025-11-14
- DOI
- https://doi.org/10.1212/wnl.0000000000214388
- Citations
- 3
- Primary Topic
- Epilepsy research and treatment
- Type
- article
- Field-Weighted Citation Impact
- 4.41