HNF has been conducting patient-focused research since 2007, when it launched its first funded study, What It’s Like To Live with Charcot-Marie-Tooth (CMT): Those Who Know It Best. Conducted by Drs. Elizabeth Barrett and Carole Birdsall, the study was published as the Essential CMT Guide, to help people with CMT understand how the disease affects others and know they are not alone. It has been shared with our Centers of Excellence, at medical conferences, and as a free download to educate healthcare professionals and others about the lesser-known symptoms, emotions, and experiences of living with CMT.
Our TRIAD Program
This patient-focused research became the pillar of our Therapeutic Research In Accelerated Discovery (TRIAD) program. At HNF, patients are the key stakeholders in every aspect of therapy development. By understanding patients’ experiences, preferences, and outcomes, we can better assess and prioritize our projects and support our industry partners.
Patient-Focused CMT Summits
“Patient-centered” means the meaningful involvement of patients, caregivers, clinicians, and other healthcare stakeholders together in a summit format: topic selection, panel participation, audience Q&A, and all the conversations in between.
2022: Patient-Focused CMT THRIVE Summit (Virtual)
- Self Love & Advocacy
- CMT Research
- Kids, School & Social
- Accessibility
- Work & Career
- Family & Relationships
2021: Movement is Medicine™ Virtual Summit
- The Brain-Changing Benefits of Exercise & Mindful Meditation
- Therapy Across the Lifespan of the Disease
- Core & Balance
- Nutrition
- Strength Training
Learn more at movementismedicine.com.
2019: Movement is Medicine™ Summit (Phoenix, AZ)
- Brain and Body Rehabilitation
- Aquatic Exercise
- Nutrition
- Bracing
- Yoga
- Strength Training
- Rock Climbing
- Tai Chi
- CMT Research
- Gait Analysis Study
2018: Patient-Centered CMT Behavioral Health Summit (College Park, MD)
- Fast Forward Program with Lisa McCarthy
- Assertiveness & CMT
- Surgery Outcomes & Behavioral Health
- Nutrition & CMT
2017: Patient-Centered Summit for CMT and HNPP (Cambridge, MA)
- Chronic Pain & Social Networks
- Types of Pain & Mechanisms
- Pain Management
- Partnering with Your Healthcare Providers
- Communicating About Pain
2016: Patient-Centered CMT Summit (New York, NY)
- CMT Research
- Advocacy
- Role of Caregivers
- Nutrition
- Clinical Trials
- Patient-Reported Outcomes
Industry-Sponsored Focus Group & Patient-Reported Study
We partnered with Acceleron on a focus group to help shape the Phase II study design of ACE-083, and on a groundbreaking GRIN patient-reported study.
Patient-Focused Drug Development (PFDD) Meeting
All of these patient-focused initiatives prepared us for the externally-led Patient-Focused Drug Development (PFDD) meeting HNF held on September 28, 2018 to help the FDA and other stakeholders better understand what it is like to live with CMT. HNF has developed a regulatory strategy, with a menu of resources and tools, to support the FDA’s guidelines for advancing therapies for rare diseases.