Publications

See our presentations on Dravet syndrome and ADOA.

Dravet Syndrome Presentations

Substantial improvements in overall seizure burden and seizure-free days in patients with Dravet syndrome treated with zorevunersen: Results from Phase 1/2a and open‑label extension studies

IEC 2025

September 1, 2025

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EMPEROR Phase 3 study design: Evaluation of zorevunersen as a potential disease-modifying therapy for Dravet syndrome

IEC 2025

September 1, 2025

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Zorevunersen demonstrates potential as a disease-modifying therapy in patients with Dravet syndrome through durable seizure reduction and improvements in cognition, behavior, and functioning with up to 36 months of maintenance dosing in open-label extension studies

IEC 2025

August 31, 2025

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Zorevunersen demonstrates potential as a disease-modifying therapy in patients with Dravet syndrome through durable seizure reduction and improvements in cognition, behavior, and functioning with up to 24 months of maintenance dosing in open-label extension studies

EPNS 2025

July 10, 2025

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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

AES 2024

December 6, 2024

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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

AES 2024

December 6, 2024

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Small changes on the Vineland-3 are meaningful to caregivers of patients with Dravet syndrome

AES 2024

December 6, 2024

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Spectral EEG analysis demonstrates decreased slow-wave activity in patients with Dravet syndrome after treatment with zorevunersen (STK-001), an antisense oligonucleotide

AES 2024

December 6, 2024

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24-Month Analysis of BUTTERFLY A Prospective, Observational Study to Investigate Seizures and Comorbidities in Children and Adolescents with Dravet Syndrome

EEC 2024

September 10, 2024

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ADOA Presentations

Flavoprotein fluorescence in the 24-month FALCON natural history study of autosomal dominant optic atrophy: A potential biomarker for mitochondrial dysfunction

ARVO 2025

May 3, 2025

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FALCON: A prospective natural history study in patients with OPA1-autosomal dominant optic atrophy (ADOA)

NANOS 2025

March 15, 2025

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FALCON: A Prospective Natural History Study of Patients with OPA1-Autosomal Dominant Optic Atrophy (ADOA)

ARVO 2024

May 6, 2024

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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)

ARVO 2024

May 6, 2024

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Mitochondrial Dysfunction in Autosomal Dominant Optic Atrophy (ADOA) Assessed in FALCON, A Non-interventional, Natural History Study

ARVO 2024

May 6, 2024

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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)

ASGCT 2022

May 16, 2022

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Models of Autosomal Dominant Optic Atrophy (ADOA) using iPSCs and Response to Targeted Augmentation of Nuclear Gene Output (TANGO) Antisense Oligonucleotides (ASOs) Treatment

ARVO 2022

May 2, 2022

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Antisense oligonucleotide mediated increase in OPA1 improves mitochondrial function in fibroblasts derived from patients with autosomal dominant optic atrophy (ADOA)

ARVO 2021

May 4, 2021

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Antisense oligonucleotide mediated increase of OPA1 expression using TANGO technology for the treatment of autosomal dominant optic atrophy

ARVO 2020

June 12, 2020

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Antisense oligonucleotide mediated increase of OPA1 expression using TANGO technology for treatment of autosomal dominant optic atrophy

ASGCT 2020

May 12, 2020

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Featured Publications

Antisense oligonucleotides increase Scn1a expression and reduce seizures and SUDEP incidence in a mouse model of Dravet syndrome

Science Translational Medicine

August 26, 2020

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Antisense oligonucleotide modulation of non-productive alternative splicing upregulates gene expression

Nature Communications

July 9, 2020

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