Help for Today and Hope for Tomorrow: Empowering the CMT Community at the 2024 CMTA Patient & Research Summit in Denver
The 2024 CMTA Patient & Research Summit in Colorado was a remarkable gathering of more than 450 attendees—both in person and virtually—making it one of the largest CMTA Summits to date. Held from September 6-8 in Denver, the event welcomed participants from across the US, six other countries, and virtual attendees from 10 countries, with over 60% attending for the first time.
CMTA CEO Sue Bruhn, PhD, captured the essence of the weekend: “The CMTA Patient & Research Summit embodied our core focus to provide Help for Today and Hope for Tomorrow. Beyond the practical solutions for managing everyday life with CMT, it created a unique space for attendees to connect, share, and build lasting relationships with fellow community members, exhibitors, and speakers.”
A Space to Connect and Belong
From the moment attendees arrived on Friday and participated in a “dine-around” across Denver’s vibrant restaurant scene, the sense of community was undeniable. For many, this was their first experience meeting others with CMT face-to-face—a powerful reminder that they are not alone in their journey. One attendee captured this sentiment perfectly: “I came away with an improved understanding of and hope for current research initiatives, ways to live with CMT, and a profoundly increased feeling of connection to the CMTA Community.”
Saturday’s sessions were packed with educational presentations from CMT specialists, clinicians, and fellow community members. Topics ranged from the latest in symptom management to community perspectives on navigating life with CMT. Sunday’s focus shifted to research, with world-renowned CMT scientists sharing groundbreaking advancements in potential CMT treatments and ongoing projects aimed at finding a cure.
Help for Today
The Summit provided attendees with many practical tools and strategies for managing life with CMT. Expert-led sessions on physical and occupational therapy emphasized daily activities that can significantly improve quality of life. In-depth discussions on bracing options and surgical interventions offered guidance on how to approach mobility challenges and when to consider corrective foot surgery for CMT.
In addition, respiratory care and emotional well-being sessions addressed the broader aspects of living with CMT, offering solutions for managing neuromuscular breathing difficulties and mental health challenges. These useful, day-to-day strategies highlighted CMTA’s commitment to providing help for today, equipping attendees with resources they can use today to improve their lives.
Hope for Tomorrow
On Sunday, attendees learned about the significant achievements in CMT research through the CMTA’s Strategy to Accelerate Research (STAR) program. With more than 50 active research projects, including 35 sponsored initiatives, STAR has invested $30 million to accelerate research in treatments for all forms of CMT. This year alone, 9 new projects were approved, targeting both demyelinating and axonal forms of CMT. The collective impact of this research is moving us closer to treatments that will benefit the entire CMT community. One of the standout achievements shared was the rapid progress of CMT-SORD research.
Discovered through CMTA’s ongoing genetic discovery initiative, CMT-SORD advanced from gene discovery to a Phase III clinical trial in just two years, thanks to CMTA’s commitment to ensure the infrastructure was in place to start trials as soon as the science allowed. CMTA-STAR Alliance Partner Applied Therapeutics, which is conducting the trial, reported encouraging 12-month data earlier this year, fostering hope for the entire CMT community.
Breakout sessions provided updates on research for demyelinating CMT, axonal CMT, and undiagnosed forms of CMT (unknown genetic cause), highlighting the expansive reach of CMTA-STAR’s funded research. These sessions reinforced CMTA’s commitment to accelerating treatments that will bring hope to the entire CMT community. Attendees left confident, knowing that every project supported by CMTA brings us closer to effective treatments for all CMT types.
Keynote Spotlight: Vittorio Ricci
Sunday’s keynote speaker, 26-year-old Vittorio Ricci, provided an inspiring example of the power of community and advocacy. Diagnosed with the CMT-SORD in 2021 after years of uncertainty, Vittorio shared his journey—from his initial diagnosis at age 14 to receiving genetic confirmation to his involvement in a Phase III clinical trial for govorestat, a potential first-ever treatment for CMT.
Vittorio spoke passionately about how CMTA has connected him with others facing similar challenges and empowered him to actively advocate for the CMT community. He has been working closely with Applied Therapeutics on FDA outreach, advocating for accelerated approval of the company’s experimental drug for this unique type of CMT, govorestat. His story underscored the importance of family, community, and collective efforts in driving progress toward treatments for CMT.
Personal Stories of Impact
For many, the Summit wasn’t just an event; it was an emotional turning point. Clarissa, diagnosed with CMT-SORD at 18, described her experience in Denver as nothing short of transformative. Reflecting on the summit, she said, “It is an otherworldly experience to be in a room full of people with CMT. There is an unspoken bond that transcends blood… Strangers instantly turn into friends.”
Her words capture the unique sense of belonging attendees felt during the Summit. This year’s gathering provided a space where people could connect, share their experiences, and walk away with newfound friendships, all while gaining valuable knowledge about the future of CMT research and managing their disease.
A Special Thank You to Our Sponsors and Exhibitors
CMTA would like to extend our heartfelt gratitude to the sponsors and exhibitors of the 2024 Patient & Research Summit. Your generous support and participation made this event possible and helped bring the CMT community together for a weekend of learning, connection, and hope.
Acceleration Sponsor:
Applied Therapeutics
Summit Sponsors:
ActioBiosciences, ArmatusBio, PsychoGenics, Nervosave Therapeutics, NMD Pharma, and Novartis
Corporate Sponsors:
Allard USA and TurboMed Orthotics
Exhibitors:
All Bodies Community, Cadense, Evergreen Prosthetics and Orthotics, MotiveLabs, Organileaf Farms, Ottobock, Rise Prosthetics + Orthotics, SitnStand, and Variantyx
Your commitment to accelerating CMT research, supporting the community, and fostering innovation is invaluable. Together, we are making meaningful strides towards a world without CMT. Thank you for being an integral part of our mission.
Looking Ahead
The weekend wasn’t just about reflecting on progress but was about looking towards the future. With more than half of the attendees being first-timers, there was an undeniable momentum in the air—a growing movement united by the goal of finding treatments and, ultimately, a cure for CMT. As one attendee said, “The Summit, time and time again, demonstrated the power of a community and an advocacy organization to make meaningful impacts.”
As we look ahead to next year’s Summit, scheduled for September 5-7, 2025, in Indianapolis, Indiana, we remain committed to providing Help for Today and Hope for Tomorrow. Building on this year’s success, we will continue to bring even more members of the CMT community together for another unforgettable weekend.
Published on: October 3, 2024