September 16 & 17th
Friday, September 16th
- Dr. Chris Austin, founding director of NCATS and CEO of Flagship Pioneering.
- Dr. Vera Fridman, University of Colorado
- Metabolic coupling between myelinating Schwann cells and axons- Elisabetta Babetto, University at Buffalo
- SORD deficiency- Andrea Cortese, University College London
- Mitochondrial dysfunction and axon transport- Alexander Van Der Bliek, UCLA
- SARM1 in CMT1A- Kate Moss, Johns Hopkins University
- Gene replacement therapy for inherited neuropathies- Rachel Bailey, UTSW
- ASOs for CMT1A
- CMT2S/SMARD1 clinical trial- Shibi Likhite, Nationwide Children’s Hospital
- Directed evolution for AAV targeting- Mohammadsharif Tabebordbar, Harvard University
- Macrophages in demyelinating CMT- Rudolf Martini, University of Würzburg
- Modulating the unfolded protein response- Maurizio D’Antonio, San Raffaele Scientific Institute
- Mitofusin activators for CMT2A- Gerald Dorn, Washington University in St. Louis
- 4. PIKfyve inhibition for CMT4B1 and related neuropathies- Alessandra Bolino, San Raffaele Scientific Institute
- Enhancing autophagy for CMT1A- John Blackwood, Samsara Therapeutics
- Muscle fat fraction
- Neurofilament light
- An international collaboration to discover CMT biomarkers (NCAM1 and GDF15)- Matt Jennings, University of Cambridge
For the CMT Patient Community
Saturday, September 17th
Dr. Keith Fargo, CMT Research Foundation
Dr. David Herrmann, University of Rochester
Dr. Herrmann will discuss the most groundbreaking scales used to measure CMT and its progression. He will discuss their relevance to clinical trials and why it is critical that patients participate.
Drs. Marina Kennerson, Wolfgang Pernice, Jim Lupski, Vincent Timmerman, Stephan Zuchner, Andrea Cortese, Tony Antonellis
Drs. Kennerson and Pernice will lead a discussion with the world’s leading CMT geneticists, including the two scientists who found the first CMT gene in 1991.
Melissa DuPont, Sanofi
Ms. DuPont is the patient engagement lead at one of the world's biggest pharmaceutical companies. She will discuss how clinical trials are designed and how pharma need patient input.
- Other types of CMT
- Pediatric Considerations
- Bracing Considerations
- Patients getting Involved
Dr. Mario Saporta, University of Miami
Dr. Saporta will discuss the current clinical trials and their progress. He will also share the planned clinical trials coming soon for CMT.
Dr. Paul August (Agios Pharmaceuticals), Dr. Arthur Suckow (DTx Pharma), Dr. Scott Harper (Armatus Bio), Dr. Thomas Pedersen (NMD Pharmaceuticals), Dr. Chris Lorson (Shift Pharma), Dr. Sylvain Celanire (Augustine Therapeutics)
Dr. August will lead a discussion with CEOs and scientists from biotechs working in CMT. They’ll discuss why CMT is the next indication of interest for many companies and investors.
Dr. Keith Fargo and Susan Ruediger will discuss how we, as a community, can continue to push the momentum of CMT into treatments and cures.
The final hour of the day is dedicated to giving each attendee time to talk with the scientist who presented throughout the day. Attendees will have one-on-one time to ask questions most relevant to them.
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.