Antisense oligonucleotides increase Scn1a expression and reduce seizures and SUDEP incidence in a mouse model of Dravet syndrome August 26, 2020 Source Science Translational Medicine
Antisense oligonucleotide modulation of non-productive alternative splicing upregulates gene expression July 9, 2020 Source Nature Communications
Zorevunersen (STK-001) demonstrates potential for disease modification, including reductions in seizures and improvements in cognition and behavior in children and adolescents with Dravet syndrome December 6, 2024 Source AES 2024
Patients with Dravet syndrome in open-label extension studies of zorevunersen (STK-001) have durable reductions in seizure frequency and ongoing improvements in cognition and behavior December 6, 2024 Source AES 2024
Small changes on the Vineland-3 are meaningful to caregivers of patients with Dravet syndrome December 6, 2024 Source AES 2024
Spectral EEG analysis demonstrates decreased slow-wave activity in patients with Dravet syndrome after treatment with zorevunersen (STK-001), an antisense oligonucleotide December 6, 2024 Source AES 2024
FALCON: A Prospective Natural History Study of Patients with OPA1-Autosomal Dominant Optic Atrophy (ADOA) May 6, 2024 Source ARVO 2024
OSPREY: An Open-label Study to Investigate Safety, Tolerability, and Exposure of the Antisense Oligonucleotide (ASO) STK-002 in Patients with OPA1 Autosomal Dominant Optic Atrophy (ADOA) May 6, 2024 Source ARVO 2024
Mitochondrial Dysfunction in Autosomal Dominant Optic Atrophy (ADOA) Assessed in FALCON, A Non-interventional, Natural History Study May 6, 2024 Source ARVO 2024
STK-002, an Antisense Oligonucleotide (ASO) for the Treatment of Autosomal Dominant Optic Atrophy (ADOA), is Taken Up by Retinal Ganglion Cells (RGCs) and Upregulates OPA-1 Protein Expression After Intravitreal Administration to Non-human Primates (NHPs) May 16, 2022 Source ASGCT 2022
Models of Autosomal Dominant Optic Atrophy (ADOA) using iPSCs and Response to Targeted Augmentation of Nuclear Gene Output (TANGO) Antisense Oligonucleotides (ASOs) Treatment May 2, 2022 Source ARVO 2022
Antisense oligonucleotide mediated increase in OPA1 improves mitochondrial function in fibroblasts derived from patients with autosomal dominant optic atrophy (ADOA) May 4, 2021 Source ARVO 2021
Antisense oligonucleotide mediated increase of OPA1 expression using TANGO technology for the treatment of autosomal dominant optic atrophy June 12, 2020 Source ARVO 2020
Antisense oligonucleotide mediated increase of OPA1 expression using TANGO technology for treatment of autosomal dominant optic atrophy May 12, 2020 Source ASGCT 2020