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.

[thermometer raised=10,042,000 target=10,000,000]

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.

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.

What is CMT?

Changing One Family's Legacy

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

DTx Pharma* Armatus Bio* Shift Pharmaceuticals*

Nanite Bio

Emory University

Augustine Therapeutics*

Oryzon*

Vanderbilt University

Samsara Therapeutics

Engagement With the FDA

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.

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.