global genes

HNF & Inspire Present Poster at Global Genes 2019 Rare Drug Development Symposium

by | Nov 6, 2019 | 0 comments

In June 2019, Allison Moore, Founder/CEO, HNF and Judy Chandler, Director of Partnerships, Inspire, presented the poster “Partnership and progress for a rare disease community: How Inspire and the HNF collaborated to grow the CMT patient community and accelerate research”

In 2009, HNF partnered with Inspire, a social network for health, to launch a CMT online support community. The community has grown to more than 6,000 patient and caregiver members and generated 40,000+ posts — real-world data that reveal important trends, themes, and gaps. The poster illustrates how the partnership between HNF and Inspire contributed to several research and educational initiatives

Incorporating Pain as a Novel Research and Clinical Outcome Measure 

Using pain-related conversations and resulting data from the CMT support community, HNF developed initiatives to understand the potential link between pain and CMT, including a patient-centered pain summit and a patient-reported IRB outcomes study through their patient registry, Global Registry for Inherited Neuropathies (GRIN). Pain is now a clinically relevant endpoint in CMT treatment and key outcome measure in CMT clinical trials.

Understanding Psycho-Social Components of CMT

HNF leveraged data and content from the CMT support community about the psycho-social components of the disease to develop questions for a Natural History Study and to plan an FDA Externally-led Patient Focused Drug Development (EL-PFDD) Meeting and a Patient-Centered Behavioral Health Summit.

Capturing the Patient Voice

HNF and Inspire collaborated to use voice-activated technology to capture the CMT patient experience. Results were shared at the EL-PFDD Meeting to help HNF describe the true burden of living with CMT and how patients view the risks and benefits of CMT treatments to inform the FDA, drug developers, and other key stakeholders.

Developed CMT Healthcare Provider Directory

In 2016, Inspire collaborated with HNF to design and analyze a healthcare provider (HCP) survey in the CMT support community. The survey received 225 responses and resulted in a national directory now over 1,500 providers across many disciplines who understand how to manage patients with CMT.

Inspire and HNF were uniquely positioned to create a successful, innovative partnership beyond the critical work of peer-to-peer support for patients and families affected by CMT.



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Related Blog Posts

Building Awareness on CMT and Supporting the Patient Community – HNF Announces Support from Pharnext

HNFpharnext recently entered into a partnership with the French biopharmaceutical company, Pharnext, to help raise awareness of Charcot-Marie-Tooth (CMT) disease and support the CMT patient community through several initiatives. Building awareness is key! Pharnext’s support will assist HNF in distributing HNF’s CMT Update quarterly newsletter, enhancing the Global Registry for Inherited Neuropathies (GRIN), setting up activities for CMT September Awareness Month in the US and strengthening the CMT Inspire Community.

Quality of Life and CMT Research

Did you know that 95% of clinical trials fail? There are multiple causes, most related to efficacy or safety, which obviously can be harmful and risky for patients. The risk-reward of enrolling in trials is a judgment call based on the devastating effects of disease related to quality of life (QoL) or life-threatening disease. With CMT, the risk-reward is more of a challenging question for many, as CMT in most cases is non-fatal.

Help us answer questions that your doctors and the CMT Research Community aren’t too sure about.

Did you know that you can become part of a community in therapy development and further research for all forms of CMT and inherited neuropathies? The mission of the Global Registry for Inherited neuropathies (GRIN) is to collect clinical and genetic information from patients with ALL forms of Charcot-Marie-Tooth (CMT) and other related rare and ultra rare inherited neuropathies.

Hot off the press

It seems like almost weekly there is another new publication on CMT with interesting basic biology. While an earlier “Hot off the press” highlighted the work of Cherry and co-workers at the University of Texas Southwestern Medical Center who showed show that neurons lacking a gene for rab7 result in neuropathy.

New Study on CMT Type 2B

If we are to learn more about CMT and the effectiveness of rehabilitation it is worth asking the patient and their caregiver. A recent Italian study by Padua et al recently described a survey of CMT patients and caregivers and their perspectives and perceptions of rehabilitation efficacy and needs.

Allison Moore is going to be published!

HNF’s CSO (Chief Scientific Officer), Sean Ekins wrote a blog about his work with Allison Moore and her two “fighter Mom” friends Lori Sames and Jill Wood. He named his blog: “Rare disease heroes – Extraordinary collaborators we should be listening too.” Sean helped my friends and I write a paper called: “Multifaceted roles of ultra rare and rare disease patients/parents in drug discovery.” YES, Allison Moore is going to be published! It will be in Drug Discovery Today, soon. The link to the reprint is below.

HNF-WVU-NIOSH Study update:

We are pleased to update our readers on the progress of the WVU-NIOSH study, “A Bi-Directional Translational Model of Exercise Training in the Treatment of Charcot-Marie-Tooth Disease”

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