CMT Endgame - Ending The Legacy of CMT1A

It’s time to put an end to CMT1A.

The suffering cannot persist any longer.

We envision the route towards clinical trials, treatments and ultimately, a cure.

Join us as we embark on this journey.

Together, let’s raise $10 million to advance treatments and find a cure for CMT1A.

There is now a $100,000 match for any donations towards ENDGAME

The CMT Research Foundation is registered as a 501(c)(3) nonprofit organization. Contributions are tax-deductible to the extent permitted by law.

If you would like to donate to non-CMT1A, please click here.


Created using the Donation Thermometer plugin$10,000,000Raised $7,947,455 towards the $10,000,000 target.$7,947,455Raised $7,947,455 towards the $10,000,000 target.79%
We are on a one-way trip directly to the ENDGAME FOR CMT1A. I, along with other family members, live with CMT and now I see my daughter and niece repeating the cycle. It just has to stop.

The CMT Research Foundation has a strategy called ENDGAME. It is working. We are closer than ever to finding treatments and cures.

$10 million will fund the right research to develop treatments for CMT1A.  For the first time in more than 45 years, I am encouraged that the science is at a point where treatments and cures are soon to be a reality.

We can do this together.  We WILL cure CMT1A, then use the knowledge and science to cure ALL types of CMT.

Peter deSilva
Board Member & Campaign Chair

Former President – Retail of TD Ameritrade
Fellow, Harvard University Advanced Leadership Initiative

CMT1A: A Debilitating Disease with No Treatments Available, Yet.

3 Reasons Why We’re at a Critical Point:

  • Gene therapies are rapidly coming online, and new genomic testing capabilities are available.
  • Pharmaceutical companies are now interested and eager to develop treatments for CMT1A.
  • Companies new to CMT, in partnership with the CMT Research Foundation, have real power to catalyze and accelerate progress.
A duplication of one gene causes CMT1A
When the duplication is silenced in mice with CMT1A, symptoms are reversed
Similar gene silencing techniques are already approved for other diseases
There are no treatments for CMT1A
CMT1A is the most common type of CMT, affecting almost 1.5 million people worldwide
Its prevalence makes CMT1A an attractive target for pharmaceutical companies

Why the CMT Research Foundation: We Make Advances Happen

The CMT Research Foundation’s Scientific Advisory Board identified five key priorities to end CMT. To date, 9 projects have been funded for CMT1A, with 5* of those preparing for clinical trials.


Catalyzing Gene Therapies
Bypassing the Blood-Nerve Barrier
Leveraging Adjunct Therapies
Unleashing the Power of Researchers
Accelerating Clinical Trials

Why We Are Taking This Approach: Two Important Success Stories

Turning $128,000 Into $1 Billion for CMT Drug Development

Novartis acquires DTx Pharma: a company that CMTRF introduced to CMT.

Starting in Academia, Moving to Biotech

Through CMTRF funding, world-renowned CMT scientist and clinician Dr. Kleopas Kleopa built a genetic therapy for CMT1A. It’s now licensed by Armatus Bio.

Armatus Bio CMT

Why Your Support is Needed: Imagine the Possibilities

Donors are the catalysts for the early proofs of concept to get us to our ENDGAME.


  • Patient funding of projects, especially those that unlock possible new solutions, is a powerful incentive for pharmaceutical industry partners with deep pockets to make additional direct investments.
  • Donor dollars support CMT Research Foundation’s early funding of pre-clinical proof of concept studies that can result in new interest from new researchers.
  • CMT Research Foundation shares in the profits from commercial success, then reallocates those dollars to additional projects until all forms of CMT are conquered.


For more information, or to schedule a presentation, please contact Susan or Anna.

Susan Ruediger

Co-Founder, Chief Mission Officer 678-595-2817 [email protected]

Anna Combes

VP of Philanthropy
[email protected]


4062 Peachtree Road
Suite A209
Atlanta, GA 30319

Phone Number


Media Inquiries

George Simpson


[email protected]