Research Day – Friday, April 25, 2025
7:45am: Breakfast & Vendor Tables
8:45am: Welcoming & Opening Remarks
9:00am: Keynote: Thomas Holm Pedersen, PhD, MSc, CEO, NMD Pharma
9:30am: Patient Voices | Why We Need Clinical Trials Now
Speakers: Reagan Warren, CMT1A | Lindsey Flynt, CMT6 Mom, MTRFR/C12orf65, Alana Kohler, CMT4A
Hear powerful stories from CMT patients highlighting the urgent need for clinical trials. Discover how these trials pave the way for treatments and cures, and learn how your participation can make a life-changing impact.
10:15am: Panel: What It’s Like: Patients in Natural History Studies, CMT Trials, & Breakthroughs
Speakers: Jenneen DeFiore, Thermo Fisher Scientific | David Washabaugh, CMT1B | Gretchen Cappiello, CMT1A, HNF Board | Joshua Burns, PhD FFPM RCPS (Glasg) FAHMS, St. Jude Children’s Research Hospital | Lawrence Van Helleputte, PhD, Droia Ventures
Hear firsthand experiences from patients who have participated in natural history studies and clinical trials, alongside insights from researchers, industry leaders, and pharmaceutical representatives. Discover how their collective efforts are advancing breakthroughs, refining trial designs, and shaping the future of CMT research and care.
11:15 AM: Panel: How to Run an Effective Center of Excellence & Prepare for Clinical Trials
Speakers: Bridget McGowan, MD, Sara Beyler, CO, LO, Rachel Galant, MSNM, OTR/L, Lurie Children’s Hospital | Katy Eichinger PT, PhD, U of Rochester | Charles K. Abrams, MD, PhD, University of Illinois Health
Join trailblazers in the field as they share strategies for building impactful Centers of Excellence. Learn best practices for patient care, fostering collaboration, and achieving clinical trial readiness to drive breakthroughs in CMT research and treatment.
12:15pm: Lunch & Vendors
Take a break to enjoy lunch and connect with fellow attendees. Vendor and sponsor tables will be open, showcasing the latest CMT research, resources, and innovations.Don’t miss this opportunity to explore and engage!
1:15pm: Presentation: Will My CMT Subtype Qualify for Therapeutics? How 1 Subtype Impacts All.
Speakers: Matt Jarpe, PhD, HNF Research Consultant, Vera Kiyasova, MD, PhD, NMD Pharma, Gideon Sharpiro, PhD, Founder & CSO, Miralinc | Ryan Castoro, DO, MS, University of Missouri School of Medicine
Explore how HNF is advancing innovative research approaches to connect CMT subtypes and prepare for basket trials. Learn how these efforts are accelerating therapeutic development, ensuring broader treatment eligibility, and demonstrating how progress in one subtype benefits all CMTs
2:00pm: Presentation: Matilda, One in a Million,
Speaker: Natalia Salejko, HNF Board
2:10pm: Presentation: A Peek Inside a CMT Research Laboratory,
Speakers: Dianna Willis, PhD, Burke Neurological Institute & Weill Cornell Medicine
Join Dr. Dianna Willis for an exclusive look into the groundbreaking research advancing treatments for CMT. Learn how her team’s work on knockout rat models is driving progress in gene replacement therapy and HDAC6 inhibitors. Discover how these findings have the potential to support multiple CMT subtypes, paving the way for transformative, cross-subtype breakthroughs in care.
2:45pm: Presentation: CMT Deploy, Our 3-Year Vision & Beyond
Speakers: Allison Moore, CEO, HNF | Eric Bolling, HNF Board | Laurie Kirkegaard, NPL Impact Agency
Get a front-row seat to HNF’s bold vision for transforming CMT research and care! Discover how the groundbreaking CMT DEPLOY strategy is revolutionizing clinical trials, advancing innovative therapies, and driving real progress for all CMT subtypes.
3:15pm: Break
3:30 PM: Fireside Chat: Biomarkers in Action: How the Biobank is Driving CMT Solutions
Speakers: Terry Jo Bichell, PhD, MPH, CEO, COMBINEDBrain | Bruce Carter, PhD, Vanderbilt School of Medicine | Michael Imperiale, MD, CMO, Actio Biosciences | Daniel Claassen, MD, MS, Vanderbilt University Medical Center
Explore the critical role biomarkers play in advancing CMT therapies and achieving FDA approval. Discover how patient-donated samples are driving breakthroughs in biomarker research and shaping the future of clinical trials. Learn about the impact of HNF’s Biobank, onsite at the summit, and engage with experts in this interactive session.
4:30pm: Presentation: CMT Tech Frontier – Digital Wearable Study Phase 2 & Results
Speakers: Kayla Cornett, PhD & Marnee McKay, PhD, U of Sydney | Tina Duong PT, PhD, Stanford School of Medicine | Tim Estilow, OTR/L, CHOP | Florian Thomas, MD, MA, PhD, MSc, Hackensack U Medical Center
Join Dr. Kayla Cornett and the HNF team as they unveil Phase 2 results from their groundbreaking wearable tech study with BioSensics. See how real-world mobility data is transforming clinical trials, advancing biomarkers, and accelerating CMT treatments—while revealing how everyday factors like footwear, flooring, and clinician training impact outcomes.
5:15pm: C-Path & Closing Remarks
6:00pm: Vendors & Networking
Research Day – Saturday, April 26, 2025
7:45am: Breakfast & Vendor Tables
8:45am: Welcoming Remarks
9:00am: CMT Speed-friending – Break the ice and kick off your day by connecting with fellow attendees in this fast-paced and interactive session! Exchange introductions, share laughs, and build new friendships in a lively, fun-filled atmosphere.
9:30am: CMT Recharge – Start your day with relaxation and renewal in this stretch, meditation, breathwork, and sound bath session led by NASM-certified experts Julie Stone and Shannon Johnson. Designed for individuals with CMT, this workshop promotes balance, stress relief, and overall well-being.
10:15am: CMT Pelvic & Posture – Discover how pelvic alignment & posture impact mobility and daily life with CMT. Join NASM-certified experts Julie Stone & Shannon Johnson for a movement workshop tailored to the unique challenges of CMT for improving comfort and function.
11:00pm: Alleviating CMT Pain & Deformities in Adults and Kids with Surgery
Speaker: CMT Expert Surgeon Dr. Glenn Pfeffer of Cedars Sinai explores surgical options for pain relief and foot deformity correction, covering advancements, candidacy, and recovery insights. *Available for one-on-one consults Friday (9 AM – 5 PM) and Saturday (until 10:30 AM).
11:10am: CMT Pain Management & Fighting Fatigue – Share your experiences with CMT-related pain and fatigue. Explore effective strategies to manage discomfort and boost energy with practical tools and insights to help you feel empowered and in control.
12:10pm: Nutrition, CMT & Health: Eat to Beat Fatigue – Learn why the intersection of what, when, & how much we eat not only improves overall health, but also improves energy, pain, & mood when living with CMT.
Speaker: Melissia Schmidt, PN2-MHC, M.S.Ed, MindSet Nutrition Coaching, LLC
1:00pm: Lunch & Vendors – Take a break to enjoy lunch and connect with fellow attendees. Vendor and sponsor tables will be open, showcasing the latest CMT research, resources, and innovations.
2:00pm: CMT, Be Boldy You – Explore strategies to nurture your mental and emotional well-being while navigating life with CMT. This empowering workshop offers tools to build resilience, boost self-confidence, and embrace your authentic self with boldness and positivity.
3:00pm: Why Join Team CMT? – Discover the power of community and advocacy with Team CMT! Learn how joining this global movement supports CMT research, raises awareness, and connects you with others making a difference in the fight against CMT.
3:20pm: CMT, Test & Try Bracing & Mobility Aids – Test out and explore lots of options for bracing and mobility aids such as crutches, canes, wheelchairs, scooters, and more! Participate in hands-on demos, ask questions, and discover the best solutions to enhance your mobility, comfort, and independence.
4:45pm: Fashion & Adaptive Clothing Show – Join us for an interactive adaptive fashion showcase! Attendees will have the opportunity to show off their favorite pieces, including AFO- friendly shoes, easy-fastening garments, and other innovative designs. Celebrate style, function, and creativity while discovering how adaptive fashion meets every day needs with flair.
5:15pm: Closing Remarks – Wrap up the day with inspiring reflections and key takeaways from the summit. Celebrate the connections made, the knowledge shared, and the progress toward a brighter future for the CMT community.
5:30pm: Dessert Party & Raffle Prizes!
*Winners will be announced & dessert will be served
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