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SYNGAP1 Conference 2022

Registration: syngap.fund/treat

EVENT DETAILS

Join us for the SynGAP Research Fund’s SYNGAP1 Conference 2022: Charting Our Rare Disease Treatment Path

Professional Meeting: Thursday, December 1

Family Connect: Friday, December 2

Who: SYNGAP1 families, doctors, scientists, researchers, & industry reps

What: A two-day conference, with scientific meeting (Thursday, December 1) & family meeting (Friday, December 2)

When: December 1st & 2nd, 2022

Where: Holiday Inn Express & Conference Center, 920 Broadway, Nashville, TN, 37203

🏨 Book a room at the HIE using our group rate of $199

Why: The American Epilepsy Society (AES) is the annual meeting epilepsy conference to attend. SRF will be holding a SYNGAP1 scientific meeting the day prior to the first day of AES for SYNGAP1 parents, scientists, researchers, & other industry. We’ve added a special non-science family meeting the following day!

Sponsorship Opportunities are Available

Contact us at info@syngapresearchfund.org for sponsorship opportunities.

AGENDA

Scientific Meeting

Thursday, December 1 from 8am-5pm

The conference is intended to bring rare disease stakeholders across the research and regulatory spectrum together to discuss research interventions of interest to the SYNGAP1 community and develop a road map for next steps in trial design for therapeutics for SYNGAP1. Audience members are expected to include SYNGAP1 parents, SYNGAP1 researchers, and other rare disease parents and researchers interested in learning novel science and research regulatory aspects of rare diseases that can inform SYNGAP1 treatment development.

Tentative Agenda (subject to change)

Session #1: The State of Rare Disease Gene Based Trials. Moderator: Dr. Ingrid Scheffer, AO, MBBS, PhD (University of Melbourne)

8:10 AM- 10 AM (30 min. per speaker and 20 min Q&A)

  • 8:10: Dr. Ed Kaye, MD, (CEO and Director of Stoke Therapeutics) – Stoke TANGO precision medicine platform 
  • 8:40: Dr. Timothy Yu, MD, PhD, (Boston Children’s Hospital and Harvard Medical School) – Trials in Angelman syndrome: from N of 1 to larger trials
  • 9:10: Dr. Jeff Coller (Johns Hopkins)—mRNA Amplifier Technology for SYNGAP1
  • 9:40-10 AM Moderated discussion with panelists and audience. 

Session #2: Defining the SYNGAP1 Clinical Spectrum: Identifying Outcomes for Preclinical and Clinical Trials.  Moderator: Dr. Hans Schlecht, MD, SynGap Research Fund Science Director

  1. 10 AM-11:50 AM (30 min. per speaker and 20 min Q&A)
  2. 10 AM: Dr. Kimberly Wiltrout, MD (Boston Children’s Hospital and Harvard Medical School)—SYNGAP1 clinical spectrum from Ciitizen database 
  3. 10: 30 AM: Dr. Constance Smith-Hicks, MD, PhD (Kennedy Krieger Institute and Johns Hopkins University)—Sleep in SYNGAP1
  4. 11 AM: Dr. Christy Zigler, PhD, MSEd (Duke University)—Measures of cognitive outcome
  5. 11: 30 AM-11:50 AM Moderated discussion with panelists and audience. 

LUNCH: 12-1 PM

Session #3: Key Efforts within SynGAP Pre-clinical Models. Moderator Dr. Rick Huganir (Johns Hopkins University)

1 PM-3:10 PM (30 minutes per speaker, Q&A 10 min)

  • 1 PM: Dr. Gavin Rumbaugh, PhD (Scripps Research Institute)-Murine effect of isolated elevation of Isoform 1 alpha
  • 1: 30 PM: Dr. Peter Kind, PhD (University of Edinburgh)—Rat model systems of SYNGAP1
  • 2 PM: TBD
  • 2: 30 PM: Dr. Elizabeth A. Heller, PhD (University of Pennsylvania Perelman School of Medicine)-epigenetic regulation of SYNGAP1-related intellectual disability
  • 3 PM-3:10 PM Moderated discussion with panelists and audience. 

Session #4: Designing a SYNGAP1 Trial. Moderator: Dr. Annapurna Poduri, MD, MPH (Boston Children’s Hospital and Harvard Medical School)

3:10 PM-5 PM (30 min per speaker, Breakout session 30 min, Moderated large group session 20 min)

  • 3:10 PM: Dr. Jacqueline French, MD (NYU) --Review Epilepsy Trial Basics
  • 3: 40 PM: Dr. Vicky Whittemore, PhD (NINDS) – NIH Funding Mechanisms for Clinical Trials
  • 4:10 PM: Breakout Sessions to design a trial 
  • 4:40 PM: Moderated Large Group Session to review ideas from Breakout Session.

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Family Connect

Friday, December 2 from 9am-9pm

Tentative Agenda (more details to come)

Morning: News you can use How to work with SRF - volunteering, fundraising, advocacy

Afternoon: 2023 strategic planning

Evening: SRF year-end dinner

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