CNS 2020 (Virtual 49th Annual Child Neurology Society Meeting, October 2020)
- Long-Term (2-Year) Safety and Efficacy of Adjunctive ZX008 (Fenfluramine HCl) for Dravet Syndrome: Interim Results of an Ongoing Open-Label Extension Study (Sullivan, Auvin, Pringsheim et al)
- Number Needed to Treat (NNT) with Fenfluramine to Achieve a Clinically Meaningful Reduction in Convulsive Seizure Frequency in Patients with Dravet Syndrome (Sullivan, Dlugos, Nabbout et al)
- Quantifying “Clinically Meaningful Changes” in Seizure Frequency - Data from 3 Phase 3 Studies of ZX008 (Fenfluramine Hydrochloride Oral Solution) in Dravet Syndrome: Do Expectations and Views Change Over Time? (Gammaitoni, Sullivan, Dlugos et al)
WMS (World Muscle Society) Virtual Conference (September – October 2020)
- Analysis of Morbidity and Mortality in Untreated Patients with Thymidine Kinase 2 Deficiency (C. Domínguez-González, C. Garone, G. D’Souza, B. Thompson, M. Morton, J. Quan, M. Hirano)
- Collaborative model for diagnosis and treatment of very rare diseases: experience in Spain with thymidine kinase 2 deficiency (C. Paradas, C. Domínguez-González, M. Madruga-Garrido, M. Hirano, I. Martí, F. Munell, A. Nascimento, M. Olivé, J. Quan, M. Dolores Sardina, R. Martí)
- MT1621 for thymidine kinase 2 deficiency (TK2d): Mechanism of action is via mitochondrial DNA incorporation (L. S Tsuruda, D. Kenny, C. Blazquez Bermejo, G. Hardman, A. Karlsson, R. Marti Seves, Y. Camara Navarro)
- Design of A Phase 3 Prospective Clinical Study for The Treatment of Thymidine Kinase 2 Deficiency (TK2d) (M. Hirano, Q. Abu Ali, C. Dominguez-Gonzalez, A. Berardo, R. Haas, C. Konnersman, R. McFarland, G. D’Souza, S. Lakhan, J. Quan, B. Thompson, B. Cohen)
UMDF Power Surge 2020 Virtual Conference (June 2020)
- Substrate Enhancement Therapy With Deoxycytidine and Thymidine in Patients With Thymidine Kinase 2 Deficiency (M. Hirano, C. Domínguez-González, C. Paradas, M. Madruga, A. Nascimento Osorio, F. Munell, H. Mandel, T. Falik Zaccai, M. Ginzberg, G. Tal, C. Garone, S. Li, E. Barca, G. D’Souza, T. Moors, B. Thompson, J. Quan)
- VIDEO PRESENTATION: Joanne Quan Presents, “Substrate Enhancement Therapy With Deoxycytidine and Thymidine in Patients With Thymidine Kinase 2 Deficiency”
Zogenix Investor Webinar (April 2020)
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Long-Term (2-Year) Safety and Efficacy of Adjunctive ZX008 (Fenfluramine Hydrochloride Oral Solution) for Dravet Syndrome: Interim Results of an Ongoing Open-Label Extension Study (J. Sullivan, S. Auvin, M. Pringsheim, et al)
Originally scheduled for presentation at now-cancelled AAN 2020 meeting.
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Safety and Efficacy of Add-On ZX008 (Fenfluramine HCl Oral Solution) in Sunflower Syndrome: An Open-label Pilot Study of 5 Patients (E. Thiele, P. Bruno, U. Vu, et al)
Originally scheduled for presentation at now-cancelled AAN 2020 meeting.
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Long-Term (2-Year) Safety and Efficacy of Adjunctive ZX008 (Fenfluramine Hydrochloride Oral Solution) for Dravet Syndrome: Interim Results of an Ongoing Open-Label Extension Study (J. Sullivan, S. Auvin, M. Pringsheim, et al)
AES 2019 (73rd American Epilepsy Society Annual Meeting, December 2019)
- Profound Reduction in Seizure Frequency (≥75%) Leads to Improved Everyday Executive Function: Analysis From a Phase 3 Study of ZX008 (Fenfluramine HCl) in Children/Young Adults With Dravet Syndrome
- Fenfluramine in Dravet Syndrome: Re-Analysis of Study 1 Using Time-to-Event to Measure Treatment Effect
- ZX008 (Fenfluramine Hydrochloride Oral Solution) Provides Clinically Meaningful Reductions in Seizure Frequency Irrespective of Concomitant AEDs Commonly Used in Dravet Syndrome: Pooled Analysis of Two Phase 3 Trials
- The Action of Fenfluramine to Prevent Seizure - Induced Death in the DBA/1 Mouse SUDEP Model is Selectively Blocked by an Antagonist or Enhanced by an Agonist for the Serotonin 5-HT4 Receptor
- ZX008 (Fenfluramine Hydrochloride Oral Solution) in Dravet Syndrome: Comparison of Study 1 Results Using 4-Week Versus 6-Week Baseline Seizure Collection
- ZX008 (Fenfluramine Hydrochloride Oral Solution) Reduces Seizure Burden by Significantly Increasing Number of Seizure-Free Days and Time Between Seizures in Patients With Dravet Syndrome
- Caring for a Child with an Epileptic Encephalopathy: Caregiver Benefit
- Evaluation of the Acute Anticonvulsant Efficacy of Fenfluramine in Mouse Models of Acute and Chronic Seizures
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Fenfluramine HCl Significantly Reduces Frequency of Generalized Tonic-Clonic Seizures in Dravet Syndrome: Pooled Analysis From Two Phase 3 Clinical Trials
Encore
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A Phase 1, Single-Dose, Open-Label Pharmacokinetic Study to Investigate the Drug-Drug Interaction Potential of ZX008 (Fenfluramine HCl Oral Solution) and Cannabidiol
Encore
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ZX008 (Fenfluramine HCl Oral Solution) Provides Long-Term, Clinically Meaningful Reduction of Convulsive Seizure Frequency in Young (<6 Years Old) Dravet Syndrome Subjects: Analysis From a Long-Term Open-Label Study
Encore
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Relationships Between Self-Regulation and Quality of Life: Results From a Phase 3 Study of ZX008 (Fenfluramine HCl Oral Solution) in Children and Young Adults With Dravet Syndrome
Encore
48th Annual Meeting of the Child Neurology Society (CNS) October 2019
- ZX008 (Fenfluramine HCl Oral Solution) Provides Long-Term, Clinically Meaningful Reduction of Convulsive Seizure Frequency in Young (<6 years old) Dravet Syndrome Subjects: Analysis from Long-Term Open-Label Study (J. Sullivan, R. Nabbout, K. Knupp et al)
- Defining a Minimal Clinically Important Difference in Seizure Frequency Using Data from a Phase 3 Clinical Study of Add-On, Low-Dose ZX008 (Fenfluramine HCl Oral Solution) in Dravet Syndrome Patients Receiving an Antiepileptic Drug Regimen Containing Stiripentol (A. Gammaitoni, J. Sullivan, D. Dlugos et al)
- ZX008 Fenfluramine HCl Significantly Reduces Frequency of Generalized Tonic-Clonic Seizures in Dravet Syndrome: Pooled Analysis from Two Phase 3 Clinical Trials (O. Devinsky, J. H. Cross, A. Gil-Nagel et al.)
- A Phase 1, Single-Dose, Open-Label Pharmacokinetic Study to Investigate the Drug-Drug Interaction Potential of ZX008 (Fenfluramine HCl Oral Solution) and Cannabidiol (B. Boyd, S. Smith, G. Farfel et al)
- Relationships Between Self-Regulation and Quality of Life: Results from a Phase 3 Study of ZX008 (Fenfluramine HCl Oral Solution) in Children and Young Adults with Dravet Syndrome (K. Bishop, G. Gioia, P. Isquith, G. Morrison)
World Muscle Society 2019
72nd American Epilepsy Society Annual Meeting, December 2018
- 3.461 - Fintepla® (Fenfluramine HCl Oral Solution) Reduces Convulsive Seizure Frequency in Dravet Syndrome Patients Receiving an Antiepileptic Drug Treatment Regimen Containing Stiripentol: A Phase 3, Randomized, Placebo-Controlled Clinical Study
- 3.463 - Fintepla® (Fenfluramine HCl Oral Solution) Provides Long-Term Clinically Meaningful Reduction in Seizure Frequency: Results of an Open-Label Extension Study
- 3.453 - Long-Term Cardiovascular Safety of Fintepla® (Fenfluramine HCl Oral Solution) in the Treatment of Dravet Syndrome: Interim Analysis of an Open-Label Safety Extension Study
- 3.202 - What Defines “Clinically Meaningful Changes in Seizure Frequency”? Analysis of Data From a Phase 3 Clinical Study of Low-Dose Fintepla® (Fenfluramine HCl Oral Solution) in Dravet Syndrome
- 2.454 - Improved Everyday Executive Function With Fintepla® (Fenfluramine HCl Oral Solution): Results From a Phase 3 Study in Children and Young Adults With Dravet Syndrome
- 2.423 - Assessing Quality of Life in Siblings of Children With Severe Epileptic Encephalopathies: A Comparative Analysis of Sibling Self-Reports and Parental Perception of Sibling Experiences
- 2.424 - Psychosocial Concerns of Growing Up With a Sibling With a Severe Epileptic Encephalopathy: Results From the Sibling Voices Survey Identify Potential Targets for Psychosocial Assessment
- 2.406 - Caregiver Stress and Benefit: Brief scales for Caregivers of Children with Epilepsy
- 2.230 - A Specific Serotonin Receptor Is Critical in the Ability of Fenfluramine to Prevent Seizure-Induced Respiratory Arrest (S-IRA) in the DBA/1 Mouse Model of SUDEP
- Effect of Low-Dose Fintepla® (Fenfluramine HCl Oral Solution) on Seizure and Non-Seizure Clinical Outcomes in Dravet Syndrome: A Case Series of 5 Pediatric Patients Receiving Fenfluramine Through Compassionate Use Protocols
- Non-Seizure and Seizure Benefits Associated With the Use of Fintepla® (Fenfluramine HCl Oral Solution) in a Subgroup of Dravet Syndrome Patients Participating in a Phase 3 Study: Interim Findings of a Survey of Investigators
- Fenfluramine is a sigma-1 receptor positive modulator in mice
- Antiepileptic effect of the enantiomers of fenfluramine and norfenfluramine in a Dravet zebrafish model
13th European Congress on Epileptology, Vienna, Austria, August 2018
- University of Washington Caregiver Benefit Scale for Caregivers of Children with Epileptic Encephalopathy
- Siblings of Epileptic Encephalopathy Patients Are at Risk for Depression and Anxiety: Results from the Sibling Voices Survey
- Impact of Severe Rare Childhood Epilepsy on Siblings Under 18 Years of Age
- Low-dose Fenfluramine Provides Significant and Long-term Seizure Reduction in Dravet Syndrome: Update and Follow-up of the Prospective Study
21st American Academy of Neurology Annual Meeting, Los Angeles, CA, April 2018
71st American Epilepsy Society Annual Meeting, Washington, DC, December 2017
- ZX008 (Fenfluramine HCl Oral Solution) in Dravet Syndrome: Results of a Phase 3, Randomized, Double-Blind, Placebo-Controlled Trial
- Low-Dose Fenfluramine Provides Significant and Long-Term Seizure Reduction in Dravet Syndrome: Update and Follow-Up of the Prospective Study
- Low-Dose Fenfluramine Provides Significant and Long-Term Seizure Reduction in Dravet Syndrome: Update and Follow-Up of the Prospective Study
- Interim Findings from the Sibling Voices Survey: Siblings of Epileptic Encephalopathy Patients Are at Risk for Depression and Anxiety
- The Lack of Effect of Food on the Pharmacokinetics of ZX008 (Fenfluramine Oral Solution): Results of a Single-Dose, Two-Period Crossover Study
- Assessing the Impact of Caring for a Child with Dravet Syndrome: Results of a Caregiver Survey
- Fenfluramine in the Treatment of Drug-Resistant Seizures: Back-Translation Using Zebrafish and Mice
- Prophylaxis of Seizure-Induced Respiratory Arrest (S-IRA) with Fenfluramine in a Mouse Model of SUDEP
- Fenfluramine Has In Vivo Activity as a Positive Modulator of Sigma-1 Receptors
- ZX008 (Fenfluramine Oral Solution) as Adjunctive Therapy for Dravet Syndrome Seizures: A Pharmacometric Approach to Quantify Potential Drug-Drug Interactions to Support Phase 3 Dose Selection
- Development of the Caregiver Stress Scale for Caregivers of Children with Epilepsy
- A Phase 1, Randomized, Open-Label, Single-Dose, 3-Period Crossover Study to Evaluate the Drug-Drug Interaction Between ZX008 (Fenfluramine Oral Solution) and a Regimen of Stiripentol, Clobazam, and Valproate in Healthy Volunteers
National Organization for Rare Diseases and Orphan Products Breakthrough Summit, October 2017
Society of Biological Psychiatry’s 72nd Annual Scientific Convention and Meeting, May 2017:
70th Annual American Epilepsy Society Meeting, December 2016:
- Evolution of Low-Dose Fenfluramine in the Treatment of Epileptic Encephalopathies: New Understandings of the Mechanisms, Basic Science, and Clinical Data
- Dravet Syndrome Hospital Utilization and Costs: An Electronic Record Single-Center Cohort Study
- History of the Use of Low-dose Fenfluramine in Pediatric Epilepsy
- Add-on Therapy with Low Dose Fenfluramine (ZX008) in Lennox Gastaut Syndrome
- An Examination of the Mechanism of Action of Fenfluramine in Dravet Syndrome: A Look Beyond Serotonin
- Long-Term Efficacy and Safety of Low-dose Fenfluramine Treatment in Dravet Syndrome: Follow-up of the Original Patient Cohort
- Low-dose Fenfluramine Significantly Reduces Seizure Frequency in Dravet Syndrome: Update of the Prospective Study
- Fenfluramine Blocks Seizure-Induced Death in the DBA/1 Mouse Model of SUDEP
- Extrapolation of Fenfluramine Pharmacokinetics from Adults to Children: Estimating PK Exposure in Patients with Dravet Syndrome
- How Does Caring for a Child with Severe, Refractory Epilepsy Affect Lives of Caregivers?
American Conference on Pharmacokinetics, October 2016:
12th European Congress on Epileptology, September 2016:
69th Annual American Epilepsy Society Meeting, December 2015:
- History of Fenfluramine in DS AES 2015 Scientific Exhibit Two Posters
- Schoonjans CNS Original Cohort AES 2015 Scientific Exhibit Poster
- Phase 3 Clinical Development Plan AES 2015 Scientific Exhibit Poster
- Ceulemans Fenfluramine in DS Efficacy AES 2015 Poster
- Schoonjans Fenfluramine in DS Cardiac Safety AES 2015 Poster
- Sourbron Fenfluramine in Zebrafish Model of DS AES 2015 Poster
2015 Child Neurology Society Annual Meeting, October 2015:
European Paediatric Neurology Society Congress, May 2015: