HNF has been conducting patient focused research dating back to 2007 when HNF launched its first funded research study titled, What It’s Like To Live with Charcot-Marie-Tooth (CMT): The Stories of Those Who Know it Best.
The guide has been disseminated to our Centers of Excellence, at medical conferences and as a free download to educate health care professionals and others about some of the lesser known symptoms, emotions and experiences of people with CMT.
The Essential Guide
The study was published by the study investigators, Drs. Elizabeth Barrett and Carole Birdsall as an Essential Guide to help people with CMT learn more about how the disease is affecting others and that they are not alone!
Our TRIAD Program
Our patient-focused research study became the pillar of our Therapeutic Research In Accelerated Discovery (TRIAD) program.
At HNF, patients are the key stakeholders in all aspects of therapy development. By understanding patients’ experiences, preferences and outcomes, we can better assess and prioritize our projects and support our industry partners.
The TRIAD Program
Therapeutic Research In Accelerated Discovery
Patient-Focused Charcot-Marie-Tooth (CMT) Summits
“Patient-Centered” refers to the meaningful involvement of patients, caregivers, clinicians, and other healthcare stakeholders TOGETHER in a summit format.
This includes topic selection, panelist participation, audience engagement during Q&A sessions, and all the wonderful conversations in between speaker sessions and at our evening reception!
2022: Patient-Focused CMT THRIVE Summit, Virtual
- Self Love & Advocacy CMT Research
- Kids + School + Social 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
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
2018: PFDD Meeting, Externally-Led Patient-Focused Drug Development Meeting, College Park, MD
All of these patient-focused initiatives prepared us for the Externally-led Patient Focused Drug Development Meeting (PFDD) which HNF held on November 18, 2018 to inform the FDA and other stakeholders to help facilitate a better knowledge of the experience patients have living with CMT.
HNF has developed a regulatory strategy with a menu of resources and tools to support the FDA guidelines for advancing therapies for rare diseases.
2017: Patient-Centered Summit for CMT/HNPP Summit, Cambridge, MA
- Chronic pain & social networks
- Types of Pain & Mechanisms
- Pain Management
- Partnering with your HCPs
- 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 Acceleron
Focus Group – help with Phase II study design of ACE-083
GRIN Ground-breaking study with Acceleron
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Using Medical Cannabis for Managing Pain in Charcot-Marie-Tooth Disease
Using Medical Cannabis for Managing Pain in Charcot-Marie-Tooth Disease - Patient Reported Outcomes Data regarding medical cannabis use for pain relief in CMT from HNF’s CMT Patient Registry, GRIN, was analyzed and published by a team lead by Brian J. Piper, PhD and ...