Only CMT Event Conducting On-Site Research Brings Regulators, Industry, Researchers, and Patients Together
ELLICOTT CITY, MD, UNITED STATES, March 19, 2026 /EINPresswire.com/ — The Hereditary Neuropathy Foundation (HNF) will host its largest Charcot-Marie-Tooth (CMT) Summit + Health and Wellness Retreat at Turf Valley Resort, convening 250–300 patients, families, clinicians, researchers, biotech and pharma leaders, device innovators, and federal regulators to accelerate therapeutic progress for CMT, a rare neuromuscular disease affecting nearly 3 million people worldwide.
A featured session, “Moving CMT Trials at Lightning Speed: FDA’s New Framework for Rare Diseases,” will spotlight modern regulatory strategy and thoughtful rare disease trial design. Dr. Janet Woodcock, former Acting Commissioner of the U.S. Food and Drug Administration (FDA) and former Director of the Center for Drug Evaluation and Research (CDER), and co-founder of Critical Path Institute (C-Path), will participate in discussions shaping the future of CMT clinical development.
Joining the session is Dr. Collin Hovinga, Senior Vice President of Rare and Orphan Disease Programs at C-Path, underscoring the importance of regulatory science tools and qualification of Clinical Outcome Assessments (COAs) in accelerating approvals.
“Ensuring we have fit-for-purpose Clinical Outcome Assessments that reflect what is meaningful to persons impacted with rare diseases is critical to reducing risk in CMT drug development. By leveraging collaborative approaches guided by FDA guidances, we can align stakeholders and meaningfully increase the probability of success.”- Dr. Hovinga
HNF remains the only CMT Summit conducting active research onsite. The HNF CMT Biobank will collect blood and biospecimens during the Summit to support biomarker discovery and disease progression research. In parallel, HNF and BioSensics sponsored “Wearable-Based Remote Monitoring Upper and Lower Limb Function in CMT Study.” is in its third year, and is actively enrolling pediatric participants ages 8–18, to capture real-world mobility data to strengthen endpoint validation and support FDA qualification efforts for digital measures.
With more than 30 CMT sessions spanning gene therapy, artificial intelligence, biomarker development, and patient-centered innovation, the Summit uniquely integrates science, regulation, and lived experience in one collaborative environment. Two dedicated patient days run alongside the scientific program, featuring adaptive movement sessions, occupational therapy guidance, assistive technology demonstrations, and individualized bracing and surgical consultations with Dr. Glenn Pfeffer of Cedars-Sinai and Mitch Warner, CPO of Helios Bracing. Live bracing demonstrations, orthotic innovations, mobility, adaptive driving service dog showcases, along with youth and family-centered programming, ensure every generation of the CMT community is supported while gaining direct access to technologies that restore confidence and independence.
For the full session agenda and registration, visit www.cmtsummit.org
The event begins Thursday evening with the Welcome Reception featuring cocktails, hors d’oeuvres, and meaningful community connection, continuing with outdoor fire pit socials and a family-friendly movie night. The Summit concludes with a Grand Finale Adaptive Fashion Show showcasing CMT models of all ages and mobilities, inclusive brands, raffle drawings, and a joyful celebration of resilience and innovation. To apply as a model, email info@hnf-cure.org.
HNF Leadership Perspective
“The future of CMT therapies depends on alignment between science and regulation, innovation and patient voice, urgency and rigor,” said Allison Moore, Founder and CEO of HNF. “By bringing regulatory experts, C-Path, industry leaders, researchers, and families together under one roof while simultaneously conducting active research, we are not just hosting a conference. We are building the infrastructure to accelerate treatments for our community.”
About the Hereditary Neuropathy Foundation (HNF)
The Hereditary Neuropathy Foundation is a global nonprofit organization dedicated to improving the quality of life for people living with CMT disease and accelerating research toward treatments and cures. Through innovative research initiatives, patient-centered clinical trial readiness programs, regulatory engagement, and community education, HNF bridges science, industry, and patient voice to drive measurable impact in CMT drug development.
Allison T. Moore
Hereditary Neuropathy Foundation
+1 212-860-5405
email us here
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Learn more & register at www.CMTsummit.org
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