Adaptive functioning and neurodevelopment in patients with Dravet syndrome: 12-month interim analysis of the BUTTERFLY observational study January 13, 2024 Source Epilepsy & Behavior
Interim results of adaptive functioning and neurodevelopment in BUTTERFLY – An observational study of children and adolescents with Dravet syndrome November 1, 2022 Source Epilepsy & Behavior
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
24-Month Analysis of BUTTERFLY: A Prospective, Observational Study to Investigate Cognition and Other Non-seizure Comorbidities in Children & Adolescents with Dravet Syndrome (DS) December 1, 2023 Source AES 2023
MONARCH & ADMIRAL: Phase 1/2a Studies in US & UK Investigating Safety and Drug Exposure of STK-001, an Antisense Oligonucleotide (ASO), in Children & Adolescents with Dravet Syndrome (DS) December 1, 2023 Source AES 2023
SWALLOWTAIL & LONGWING: Open-Label Extension (OLE) Studies for Children and Adolescents with Dravet Syndrome (DS) who Previously Participated in a Study of Antisense Oligonucleotide (ASO) STK-001 December 1, 2023 Source AES 2023
Utilization of a Pharmacokinetic (PK) Model for STK-001 in Patients with Dravet Syndrome (DS) To Support Selection of Dosing Regimens in Clinic December 1, 2023 Source AES 2023
MONARCH and ADMIRAL Interim Analyses: Phase 1/2a Studies Investigating Safety and Drug Exposure of STK-001, an Antisense Oligonucleotide (ASO), in Children and Adolescents with Dravet Syndrome (DS) December 2, 2022 Source AES 2022
SWALLOWTAIL: An Open-Label Extension (OLE) Study for Children and Adolescents with Dravet Syndrome (DS) who Previously Participated in a Study of Antisense Oligonucleotide (ASO) STK-001 December 2, 2022 Source AES 2022
Twelve-month Analysis of BUTTERFLY: An Observational Study to Investigate Cognition and Other Non-seizure Comorbidities in Children and Adolescents with Dravet Syndrome (DS) December 2, 2022 Source AES 2022
Utilization of a Pharmacokinetic (PK) Model for STK-001 in Patients with Dravet Syndrome (DS) To Predict Pharmacologically Active Doses in Clinic December 2, 2022 Source AES 2022
Quantitative EEG Analysis in Patients with Dravet Syndrome (DS) Treated in the Phase 1/2a MONARCH and ADMIRAL Studies of STK-001, an Antisense Oligonucleotide (ASO) December 2, 2022 Source AES 2022
Interim Analyses: An Open-Label Study to Investigate the Safety and Pharmacokinetics of Single and Multiple Ascending Doses of Antisense Oligonucleotide STK-001 in Children and Adolescents with Dravet Syndrome July 11, 2022 Source EEC 2022
Interim Safety, PK, and CSF Exposure Data from the Phase 1/2a MONARCH Study of STK-001, an Antisense Oligonucleotide (ASO), in Children and Adolescents with Dravet Syndrome (DS) December 3, 2021 Source AES 2021
SWALLOWTAIL: An Open-Label Extension (OLE) Study for Patients with Dravet Syndrome (DS) who Previously Participated in Studies of STK-001 December 3, 2021 Source AES 2021
A Pharmacokinetic (PK) Model for STK-001, an Antisense Oligonucleotide (ASO), Based on Data from Non-human Primates (NHP) Enables Dose Selection in Patients with Dravet Syndrome (DS) December 3, 2021 Source AES 2021
ADMIRAL: A UK Open-Label Study to Investigate the Safety and Pharmacokinetics (PK) of Multiple Ascending Doses of Antisense Oligonucleotide (ASO) STK-001 in Children and Adolescents with Dravet Syndrome December 3, 2021 Source AES 2021
BUTTERFLY, An Observational Study to Investigate Cognition and Other Non-seizure Comorbidities in Children and Adolescents with Dravet Syndrome December 3, 2021 Source AES 2021
Observational Study to Investigate Cognition and Other Non-seizure Comorbidities in Children and Adolescents with Dravet Syndrome: Baseline Analysis of the BUTTERFLY Study December 4, 2020 Source AES 2020
Reducing the Time to Diagnosis and Increasing the Detection of Individuals with SCN1A-Related Disease Through a No-cost, Sponsored Epilepsy Genetic Testing Program December 4, 2020 Source AES 2020
Safety and Pharmacokinetics of Antisense Oligonucleotide STK-001 in Children and Adolescents with Dravet Syndrome: Single Ascending Dose Design for the Open-Label Phase 1/2a MONARCH Study December 4, 2020 Source AES 2020
Targeted Augmentation of Nuclear Gene Output (TANGO) of SCN1A Reduces Seizures and Rescues Parvalbumin Positive Interneuron Firing Frequency in a Mouse Model of Dravet Syndrome December 4, 2020 Source AES 2020
Targeted Augmentation of Nuclear Gene Output (TANGO) of SCN1A Reduces Seizures and Rescues Parvalbumin Positive Interneuron Firing Frequency in a Mouse Model of Dravet Syndrome – Oral Presentation December 4, 2020 Source AES 2020
TANGO oligonucleotides for the treatment of Dravet Syndrome: Safety, biodistribution and pharmacology in the non-human primate December 8, 2019 Source AES 2019
Targeted Augmentation of Nuclear Gene Output (TANGO) of Scn1a Prevents SUDEP in a mouse model of Dravet Syndrome December 7, 2019 Source AES 2019
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