SF0034 / OcuTerra Therap - LARVOL DELTA

Potassium Channelopathies and Precision Medicine Approaches in Epilepsy: A Systematic Review of Personalized Treatment Strategies. (PubMed, Curr Neuropharmacol) - "These studies collectively offer valuable insights into precision medicines for genetic epilepsy caused by pathogenic potassium variants. This review is essential because it informs clinical decision-making, including the selection of antiepileptic drugs, thereby supporting its integration into routine clinical care for this population. However, the low level of evidence and the heterogeneity of data from the included studies limit the review."

Journal • CNS Disorders • Epilepsy

Two KCNQ2 Encephalopathy Variants in the Calmodulin-Binding Helix A Exhibit Dominant-Negative Effects and Altered PIP Interaction. (PubMed, Front Physiol) - "A novel Kv7.2/3-selective opener, SF0034, rescued current amplitudes. Our findings show that these two Helix A variants suppress channel current density strongly, consistent with their severe heterozygous phenotypes, implicate impairment of CaM and PIP regulation in KCNQ2 encephalopathy pathogenesis, and highlight the potential usefulness of selective Kv7 openers for this distinctive pathogenic mechanism and patient subgroup."

Journal • CNS Disorders • Epilepsy