Frequently Asked Questions
What is CMT?
CMT is a genetic nerve disease with over 90 known genetic causes. It affects approximately 1 in every 2500 people. Onset can be at birth or later in life and is characterized by the progressive deterioration of peripheral nerves that control sensory information and muscle function of the foot/lower leg and hand/forearm leading to significant problems with movement, touch, and balance as it advances.
Does CMT affect children?
Yes, CMT can affect a child’s physical development and, in many cases, may require a child to wear leg braces and restrict their physical activities.
Are there any approved treatments for CMT?
There are no approved disease-modifying-therapies for CMT approved by the FDA. The CMT Research Foundation is leading on various drug development efforts to change this.
How does CMTRF spend the money it raises?
CMTRF is focused singularly on finding treatments and a cure for CMT. The Research Foundation’s impact goes beyond simply making investments. We identify key problems or gaps in progress to a cure, then aggressively find partners to attack the problems. We will work with everyone with research or technology that can help overcome these key problems or gaps, including experts in the field as well as new to CMT.
What is the overall state of research for CMT?
Here are reasons for optimism in our search for a cure:
- We understand the barriers to drug development for CMT
- We leverage new forms of gene editing
- We are approaching treatments for more than half of patients
- We build upon learnings from other diseases
What are some current CMTRF-funded research projects?
CMTRF is currently funding 12 projects (with two in the preclinical stage). Here are two stories to demonstrate how our unique approach is effective:
Introducing CMT to a biotech with relevant technology
In 2019 we knew that a key barrier in drug development for CMT, was and is the ability to deliver therapies throughout the peripheral nervous system. We researched the pharmaceutical landscape and identified DTx Pharma as a company with therapies that might be adopted to CMT drug development and introduced CMT to them. The data from the initial project was promising enough to attract an additional $350,000 from the NIH for follow on studies. Moreover, DTx was able to secure $100 million in private funding using talking points derived from meetings in which the Research Foundation team helped DTx understand the relevance of their work to the potential CMT market. We have the commitment from DTx that, with continued success in the CMT1A project, they will invest enough additional capital to conduct clinical trials and apply for FDA approval.
Building upon previous successes to broaden the impact
Similarly, renowned CMT scientist and clinician Dr. Kleopas Kleopa at the Cyprus Institute for Neurology and Genetics was investigating the use of a commonly used viral technology to deliver genetic therapies for two types of CMT, but not CMT1A. CMTRF inquired if his technology might also work for CMT1A. Two and a half years later, the therapeutic is showing strong results consistent with a viable treatment for CMT1A and is now being adopted by industry to accelerate development for potential clinical trials.
Complete list here.
How can patients and families get involved in helping CMTRF?
They can donate to CMTRF in a variety of ways, including recurring donations, annual gifts, and gifts from estates. They can also:
- Become an ambassador to spread the word about the importance of finding a cure.
- Create a personal fundraising page and raise money to support critical research
- Run a charity event with proceeds going to CMTRF
- Attend our annual Global CMT Research Convention
- Follow us and share our messages on Facebook, LinkedIn, Instagram, and Twitter!
- When they shop on Amazon, they can choose CMTRF through their SMILE program
How can my business or organization support CMTRF’s mission?
You can become a corporate sponsor of our organization and our annual Global Research Conference, fund research, or cover our operating overhead that will free more resources directly for research. Additionally, you can lend your marketing expertise to help spread awareness of our mission. You can also encourage your employees to give by matching all employee donations to the CMT Research Foundation.