Agenda

September 16 & 17th Cambridge, MA
All times listed below are in Eastern Daylight Time (EDT)

Day 1

For Researchers

Friday, September 16th

Register Now
7:00-8:00 am Breakfast
  • Complementary breakfast with coffee bar
OPENING SESSIONS
8:00-8:15 am Welcome Session
  • Cleary Simpson, CMT Research Foundation
  • Keith Fargo, PhD, CMT Research Foundation
  • Marina Kennerson, PhD, ANZAC Research Institute ad Sydney Medical School
8:15-8:45 am Keynote Address
  • Chris Austin, MD, Flagship Pioneering and Vesalius Therapeutics
8:45-9:15 am Recent Development in CMT Research
  • Vera Fridman, MD, University of Colorado
9:15-9:30 am Break

THEME SESSIONS
9:30-11:30am Metabolism and Axon Degeneration
Moderator: Edritz Javelosa, PhD, Muscular Dystrophy Association
  1. Metabolic coupling between Schwann cells and axons- Elisabetta Babetto, PhD, University at Buffalo 
  2. SORD deficiency- Andrea Cortese, MD, PhD, University College London Queen Square Institute of Neurology
  3. Mitochondrial dysfunction and axon transport- Alexander Van Der Bliek, PhD, UCLA School of Medicine
  4. SARM1 in CMT1A- Kathyrn R. Moss, PhD, Johns Hopkins University
11:30-12:30 pm Lunch Buffet
12:30-2:00 pm Genetic Medicine
Moderator: Alessandra Bolino, PhD, San Raffaele Scientific Institute
  1. Gene replacements for GAN and CMT4J – Rachel Bailey, PhD, University of Texas Southwestern Medical Center
  2. PMO ASOs for CMT1A – Christian Lorson, PhD, University of Missouri and Shift Pharmaceuticals
  3. SMARD1/CMT2S Gene Therapy: Benchside to Bedside- Shibi Likhite, PhD, Nationwide Children’s Hospital
2:00-2:30 pm Break
2:30-5:00 pm Small Molecule Approaches
Moderator: Paul August, PhD, ReviR Therapeutics
  1. Inflammation in Type 1 CMT – Rudolf Martini, PhD, University of Würzburg
  2. Modulating the unfolded protein response – Maurizio D’Antonio, PhD, Ospedale San Raffaele
  3. Mitofusin activators for CMT2A – Gerald Dorn, MD, Washington University in St. Louis and Mitochondria in Motion
  4. PIKfyve inhibition for CMT4B1 – Alessandra Bolino, PhD, San Raffaele Scientific Institute
  5. Targeting autophagy for CMT1A – John Blackwood, PhD, Samsara Therapeutics
5:00-5:30 pm Break
5:30-6:30 Biomarkers for CMT
Moderator: Tiina Urv, PhD, NIH National Center for Advancing Translational Sciences
  1. Muscle fat fraction – Jasper Morrow, MBChB, PhD, University College London Queen Square Institute of Neurology
  2. An international collaboration to discover CMT biomarkers (NCAM1 and GDF15)- Matt Jennings, PhD, Columbia University

6:30-7:00 Closing Session

Day 2

For the CMT Patient Community

Saturday, September 17th

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8:00-9:00 am Breakfast
9:00-9:20 am Kick-Off Session
  • Cleary Simpson, CMT Research Foundation
  • Pat Livney/Susan Ruediger, CMT Research Foundation
  • Keith Fargo, PhD, CMT Research Foundation
9:20-10:35 am Unlocking the Genetic Secrets of CMT

Moderators: Marina Kennerson, PhD, ANZAC Research Institute and University of Sydney; and Wolfgang Pernice, PhD, Columbia University

  • Jim Lupski, MD, PhD, Baylor College of Medicine
  • Vincent Timmerman, PhD, University of Antwerp
  • Stephan Zuchner, MD, PhD, University of Miami Miller School of Medicine
  • Andrea Cortese, MD, PhD, University College London Queen Square Institute of Neurology
  • Anthony Antonellis, PhD, University of Michigan
10:35-10:45 am Break

CLINICAL TRIALS
10:45-11:15 am Understanding Clinical Trials
  • Melissa DuPont, Sanofi
11:15-11:45 am Clinical Trial Readiness: What are Natural History Studies?
  • David Herrmann, MBBCh, University of Rochester

LUNCH AND BREAKOUT SESSSIONS
11:45-12:00 pm Break
  • Grab lunch before heading into breakout sessions
12:00-12:45 pm Breakouts Round 1
Small group discussions for each topic, led by key clinicians in CMT.  
  • Types 1A and 1B – Kathryn R. Moss, PhD, Johns Hopkins University; and David Herrmann, MBBCh, University of Rochester
  • Type 2A – Mario Saporta, MD, PhD, MBA, University of Miami Miller School of Medicine
  • Type X – In Person: Charles Abrams, MD, PhD, University of Illinois at Chicago (UIC); Virtual: Wolfgang Pernice, PhD, Columbia University; and Robert Prior, PhD, University of Bonn
  • Other Types and ‘I Don’t Know My Type’ – Reza Seyedsadjadi, MD, Massachusetts General Hospital and Harvard Medical School
12:45-12:55 pm Break
  • Switch to Round 2 Breakout Sessions
12:55-1:40 pm Breakouts Round 2
  • Genetic Testing – Marina Kennerson, PhD, ANZAC Research Institute and University of Sydney; and Wolfgang Pernice, PhD, Columbia University
  • How Can YOU Make an Impact on CMT – Chelsea Layton, CMT Research Foundation
  • Bracing and Orthotics: What You Need to Know – Ken Cornell, CO, Cornell Orthotics & Prosthetics
  • Pediatrics: Care and Research – Leslie Hotchkiss Hayes, MD, Boston Children’s Hospital and Harvard Medical School
1:40-1:50 pm Break
  • Switch back to main conference room

AFTERNOON SESSIONS
1:50-2:30 pm CMT Clinical Trials: What's New and What's Next
  • Dr. Mario Saporta, MD, PhD, MBA, University of Miami Miller School of Medicine
2:30-3:45 pm Biopharma Activity in CMT

Moderator: Paul August, PhD, ReviR Therapeutics

  • Arthur Suckow, PhD, DTx Pharma
  • Scott Q. Harper, PhD, Armatus Bio
  • Thomas Pedersen, PhD, NMD Pharma
  • Sylvain Celanire, PhD, Augustine Therapeutics
  • Christian Lorson, PhD, Shift Pharma
3:45-4:30 pm Closing Session
4:30-5:30 pm Networking With the Speakers  
5:30-7:00 pm Sunset Riverboat Cruise on the Charles River

Explore the beautiful Charles River with the team at the CMT Research Foundation and the speakers from the weekend. This is a great opportunity to get to know everyone working in CMT personally.

Seating is limited – please reach out to [email protected] to request your seat(s)!

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