Page 3 - 2019 Fall CMTA Report

W E A R E F A M I L Y…

DEAR FRIENDS,

“Family means no one gets left behind or forgotten,” actor David Ogden
Stiers once said. Those are words the CMTA lives by as we push hard
every day to make sure no one in our CMT family gets left behind or
forgotten. The goal of our Strategy to Accelerate Research (STAR) is to
develop treatments for all types of CMT and the entire CMT family.

We will have many exciting research developments to share with
you in the coming months, but this issue of The CMTA Report is
focused on the Type 2 branch of our family. Because of the crossover
involved, we think many of these developments will also apply to Types 1 and 4 (which we’ll
be updating later this year). Thanks to STAR and the support of two committed Type 2 families
who are challenging the community to match $1.5 million in donations, we have set the stage
for a number of Type 2 breakthroughs on our way to treatments for CMT. Our CMT2 strategy
covers virtually all Type 2s, including those with unknown genetic causes.
Despite the diversity of genetic causes of CMT2, there are encouraging signs that a few
types of therapy may have broad application. We have identified three major therapeutic
approaches—gene therapy, inhibition of axon degeneration and small molecule therapies—that
can be used to bring CMT2 therapies to clinical trials (and that may also apply to the unknown
and/or undiagnosed Type 2s).
The opportunities to take our research efforts to the next phase are due in large part to the
momentum we generated in 2011 with our work in 2A and 2E, developing the animal models
and other tools that attracted half a dozen pharmaceutical companies as partners. These three
strategies will require a host of ancillary efforts to reach clinical trials. In total, the CMTA will
need $5 million in the next four years to implement our Type 2 strategy. If you or a loved one
has Type 2, we are asking you to help us as we keep reaching for our goals.

Donations will be matched on a dollar-for-dollar basis, thanks to challenge gifts made
by Bob and Gail Buuck, who have been supporting the CMTA since 1997 because of the
successes they’ve seen over the years, and the Chizen family, whom you’ll read more about
on page 7.
Type 2’s time in the STAR spotlight is here. With your support and our relentless hard work
and determination to bring treatments to you, we can force a breakthrough.

Sincerely,

AMY GRAY, Chief Executive Officer

FALL 2019 THE CMTA REPORT 3