Scientific Presentations and Publications
TSC-Related Epilepsy
The small molecule simufilam dose-dependently attenuates the worsening of seizures in a mouse model of tuberous sclerosis complex
Stansley B, et al. Epilepsia. 2026;00:1–13.
DOI: https://doi.org/10.1002/epi.70227 (PMID pending PubMed indexing)
Simufilam, a small molecule, reduced seizure activity in a mouse model of focal cortical malformations and demonstrated a favorable clinical safety profile
Bordey A, Kupiec J. Poster presented at: TSC International Research Conference (TSC 2025); June 26–28, 2025; Bethesda, MD.
Filamin A inhibition reduces seizure activity in a mouse model of focal cortical malformations
Zhang L, et al. Science Translational Medicine. 2020;12(531).
PubMed: https://pubmed.ncbi.nlm.nih.gov/32075941/
DOI: https://doi.org/10.1126/scitranslmed.aay0289
Convulsive seizures from experimental focal cortical dysplasia occur independently of cell misplacement
Hsieh LS, et al. Nature Communications. 2016;7:11753.
PubMed: https://pubmed.ncbi.nlm.nih.gov/27249187/
DOI: https://doi.org/10.1038/ncomms11753
MEK-ERK1/2-dependent FLNA overexpression promotes abnormal dendritic patterning in tuberous sclerosis independent of mTOR
Zhang L, et al. Neuron. 2014;84(1):78–91.
PubMed: https://pubmed.ncbi.nlm.nih.gov/25277454/
DOI: https://doi.org/10.1016/j.neuron.2014.09.009
Prior Program in Alzheimer's Disease
Phase 3 randomized clinical trials of simufilam in mild-to-moderate Alzheimer's disease
Abushakra S, et al. Alzheimer's & Dementia: Translational Research & Clinical Interventions. 2025.