By: Susan Ruediger, CEO, CMT Research Foundation

“We’re not currently working in CMT, but we know we should be.”

“We think our drug could work for CMT, but it has never been tested.”

“CMT is definitely on our interest list for expansion, but we need to learn more about the disease.”

These are just some of the comments our CMT Research Foundation team heard during conversations we had with 14 pharmaceutical companies and biotechnology leaders during the virtual 2020 BIO International Convention in early June. This event is the world’s largest gathering of the biotech industry that helps accelerate research and development in health care and other critical industries.

CMT Research Foundation is innovating at BIO Digital

When your mission is to deliver treatments and cures for a disease that has been without them for far too long, nothing stops innovation. That was the theme of this year’s conference, which was superbly fitting as we’ve refused to allow COVID-19 thwart progress in CMT research. At BIO, we met one-on-one virtually with companies working in gene therapies, biomarker discovery and small molecules that have promising technologies that could be applicable to CMT but aren’t yet working in CMT or know about the disease. Others work on CMT but were seeking expertise to overcome barriers in their development.

Our job is to proactively challenge traditional research conventions by getting more drug development companies excited about CMT and able to see new possibilities to speed treatments to market. The result is always the same: significant aha moments that spark innovation and action.

“We’re looking for partners with expertise in CMT so we can start a CMT program.”

“We need your help identifying potential therapies for CMT which we can bring into the clinic.”

During the coming days, the CMT Research Foundation has follow-up conversations scheduled with more than half of the companies we met with, including leaders in gene therapy, genetics and drug development for diseases of the nervous system. They include companies working in multiple sclerosis, ALS, Alzheimer’s and neuropathy caused by diabetes with funding from organizations like the Gates Foundation, Fox Foundation and Alzheimer’s Research Alliance.

As a patient living with CMT, it’s truly inspiring to see pharmaceutical companies of all sizes interested in my disease, all because we’ve helped challenge them to think differently about CMT and their potential impact on the disease. This is how we started working with DTx Pharma, a company that had never previously worked on CMT but had applicable technologies we thought were promising. Today, we’re funding a project that’s looking at silencing the protein that causes CMT1A, and the project is achieving its milestones. Through intentional collaboration and innovation, we will help usher in urgently needed breakthroughs to end CMT. It’s true: Nothing stops innovation.

See all the CMT research projects we’re currently funding here.


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Susan Ruediger is the CEO of the CMT Research Foundation. She is living with CMT1A and comes from a family with six generations of CMT. She’s determined to end CMT for every future generation. Read more about Susan and why she started the Foundation.