Speakers

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Allyson Berent
Chief Scientific Officer, FAST
Chief Operating Officer, GeneTx Biotherapeutics

Day One

Tuesday December 14, 2021

7:20 pm | Live Q&A Session with Your Expert Speakers

6:20 pm | Angelman Syndrome Biomarker & Outcome Measure Consortium (ABOM): Understanding What Matters to Patients with a Heterogenous Neurodevelopmental Disorder

8:40 am | A Journey Through Drug Development for Angelman Syndrome

Anish Bhatnagar
Chief Executive Officer
Soleno Therapeutics

Day Two

Wednesday December 15, 2021

5:50 pm | Live Q&A Session with Your Expert Speakers

5:20 pm | Exploring DCCR as an Important Therapeutic Option for PWS

Audrey Thurm
Director, Neurodevelopmental & Behavioral Phenotyping Service, Office of the Clinical Director
National Institute of Mental Health, NIH

Day One

Tuesday December 14, 2021

12:50 pm | Live Q&A Session with Your Expert Speakers

11:50 am | Ways to Better Measure Neurodevelopmental Outcomes & Develop Endpoints that are Patient Centric

Barbara Bailus
Assistant Professor of Genetics
Keck Graduate Institute

Day Two

Wednesday December 15, 2021

8:40 am | Panel Discussion – Diving into Specific Therapeutic Modalities

Brian Ranes
Principal Investigator, Imaging Core
Amicus Therapeutics

Day Two

Wednesday December 15, 2021

3:50 pm | Live Q&A Session with Your Expert Speakers

3:20 pm | Preclinical Development of a Cross-Correcting Gene Therapy for CDKL5 Deficiency Disorder

Charlene Son Rigby
CEO | President & Founding Board Member
RARE-X | STXBP1 Foundation

Day One

Tuesday December 14, 2021

4:30 pm | Live Q&A Session with Your Expert Speakers

2:30 pm | Patient-Powered Approaches to Expand Enrolment & The Accessibility of Natural History Data

Charles Large
Chief Executive Officer
Autifony Therapeutics

Day One

Tuesday December 14, 2021

4:30 pm | Live Q&A Session with Your Expert Speakers

3:00 pm | Precision Medicine with Kv3 Channel Modulators: Matching Mechanism to Patient Pathophysiology

Chrisopher U Missling
President & CEO
Anavex Life Sciences

Day Two

Wednesday December 15, 2021

12:50 pm | Live Q&A Session with Your Expert Speakers

11:20 am | Utilizing Precision Genetic Medicine to Treat Rare Neurodevelopmental Diseases: Focusing on Rett Syndrome

Elizabeth M. Berry-Kravis
Professor, Co-director, Molecular Diagnostics Section of the Genetic Laboratory | Director Fragile X Clinic & Research Program; Co- Director
Rush University Medical Center | Angelman Syndrome Clinic & Research Program

Day One

Tuesday December 14, 2021

12:50 pm | Live Q&A Session with Your Expert Speakers

10:50 am | How Do We Best Design Clinical Trials for Neurodevelopmental Disorders? A Clinical Researcher’s Perspective

Greg Dillon
Senior Scientist
Biogen

Day Two

Wednesday December 15, 2021

12:50 pm | Live Q&A Session with Your Expert Speakers

11:50 am | Therapeutic Strategies for Angelman syndrome

Jennifer Panagoulias
Senior Regulatory Advisor
GeneTx Biotherapeutics

Day Two

Wednesday December 15, 2021

3:50 pm | Live Q&A Session with Your Expert Speakers

2:20 pm | Developing GTX-102, an Investigational Antisense Oligonucleotide Designed to Target & Inhibit Expression of UBE3A-AS – A Regulatory Perspective

Jeremy Levin
CEO & Chairman
Ovid Therapeutics

Day One

Tuesday December 14, 2021

9:10 am | Symptomatic vs Genetic Disease Modifying Treatments

Day Two

Wednesday December 15, 2021

6:15 pm | Chair’s Closing Remarks

8:30 am | Chair’s Opening Remarks

John Kealy
Principal Scientist, Academic Liaison Manager
Ulysses Neuroscience

Day One

Tuesday December 14, 2021

12:50 pm | Live Q&A Session with Your Expert Speakers

11:20 am | Session Reserved for Ulysses Neurosciences

Katie Clapp
President and Co- Founder
FRAXA

Day One

Tuesday December 14, 2021

8:50 am | Partnering with Rare Disease Foundations to Accelerate Treatment Development: The Fragile X Example

Kevin Pelphrey
Harrison-Wood Professor
University of Virginia

Day One

Tuesday December 14, 2021

7:20 pm | Live Q&A Session with Your Expert Speakers

6:50 pm | Increasing Sensitivity & Specificity of Outcome Measures in Trials for Neurodevelopmental Disorders Using Both Clinical Outcome Assessments & Neuroimaging Approaches

Kimberly Parkerson
Senior Medical Director
Stoke Therapeutics

Day Two

Wednesday December 15, 2021

12:50 pm | Live Q&A Session with Your Expert Speakers

12:20 pm | Developing STK-001: An Oligonucleotide Treatment for Dravet Syndrome, Treatment Beyond Seizure Control

Luke Rosen
Head of Accelerated Development & Community Engagement
Ovid Therapeutics

Day One

Tuesday December 14, 2021

4:30 pm | Live Q&A Session with Your Expert Speakers

2:00 pm | Ovid’s Patient-Driven Approach to the Rapid Development of Meaningful Medicines for Neurodevelopmental Disorders

Lynn Durham
CEO & Founder
STALICLA

Day One

Tuesday December 14, 2021

3:30 pm | Precision Medicine for Neurodevelopmental Disorders

Massimiliano Bianchi
Founder, President & CEO
Ulysses Neuroscience

Day One

Tuesday December 14, 2021

7:40 pm | Chair’s Closing Remarks

8:30 am | Chair’s Opening Remarks

Michela Fagiolini
Associate in Neurology, Associate Professor of Neurology
Harvard Medical School

Day One

Tuesday December 14, 2021

7:20 pm | Live Q&A Session with Your Expert Speakers

5:50 pm | Identification, Validation & Use of Biomarkers to Accelerate Therapeutic Evaluation in Neurodevelopmental Disorders

Mustafa Sahin
Director, Translational Neuroscience Center; Director, Translational Research Program; Rosamund Stone Zander Chair, Professor of Neurology
Harvard Medical School

Day One

Tuesday December 14, 2021

12:50 pm | Live Q&A Session with Your Expert Speakers

12:20 pm | Assessing the Timing of Treatment: Driving Better Clinical Trial Design

Pam Ventola
Associate Professor
Yale Child Study Center

Day One

Tuesday December 14, 2021

7:20 pm | Live Q&A Session with Your Expert Speakers

6:50 pm | Increasing Sensitivity & Specificity of Outcome Measures in Trials for Neurodevelopmental Disorders Using Both Clinical Outcome Assessments & Neuroimaging Approaches

PJ Brookes
Program Director, Office of Rare Diseases Research
National Center for Advancing Translational Sciences, National Institutes of Health

Day Two

Wednesday December 15, 2021

9:40 am | Accelerating Clinical Trials in Rare Diseases by Moving Beyond One Disease at a Time

8:40 am | Panel Discussion – Diving into Specific Therapeutic Modalities

Randall Carpenter
Co-Founder | CMO
Allos Pharma | Rett Syndrome Research Trust

Day One

Tuesday December 14, 2021

9:10 am | Symptomatic vs Genetic Disease Modifying Treatments

Robert Komorowski
Senior Clinical Scientist
Biogen

Day One

Tuesday December 14, 2021

7:20 pm | Live Q&A Session with Your Expert Speakers

5:20 pm | EEG as a Translational Biomarker for Angelman Syndrome

Robert Ring
Chief Executive Officer
Kaerus Bioscience

Day One

Tuesday December 14, 2021

4:30 pm | Live Q&A Session with Your Expert Speakers

4:00 pm | Targeting Rare Genetic Channelopathies of Neurodevelopment

9:10 am | Symptomatic vs Genetic Disease Modifying Treatments

Samir Koirala
Director of Research, Genetic & Neurodevelopmental Disorders Unit
Biogen

Day One

Tuesday December 14, 2021

9:10 am | Symptomatic vs Genetic Disease Modifying Treatments

Sharyl Fyffe-Maricich
Executive Director, Molecular & Cell Biology, Research
Ultragenyx

Day Two

Wednesday December 15, 2021

3:50 pm | Live Q&A Session with Your Expert Speakers

2:50 pm | Exploring the Pre-Clinical Stage of Development for UX055, an Investigational Gene Therapy Program for CDD

Steven Gray
Associate Professor
University of Texas Southwestern Medical Center

Day Two

Wednesday December 15, 2021

12:50 pm | Live Q&A Session with Your Expert Speakers

10:50 am | Rett Syndrome as a Case Example of Gene Dosage Sensitivity: miRARE-mediated Feedback Regulation as a Possible Solution

8:40 am | Panel Discussion – Diving into Specific Therapeutic Modalities

Susan Hedstrom
Executive Director
Foundation for Prader-Willi Research

Day One

Tuesday December 14, 2021

9:00 am | The Role of Patient Advocacy Groups in Advancing the Development of Meaningful Treatments for Rare Disease Communities

Theresa Strong
Director of Research Programs
Foundation for Prader- Willi Research (FPWR)

Day Two

Wednesday December 15, 2021

5:50 pm | Live Q&A Session with Your Expert Speakers

4:50 pm | Gene Activation Approaches in Prader-Willi Syndrome

Yael Weiss
Vice President Business Development
Ultragenyx

Day One

Tuesday December 14, 2021

9:10 am | Symptomatic vs Genetic Disease Modifying Treatments