Page 3 - 2020 Summer CMTA Report

A M E S S A G E F R O M T H E C E O

DEAR FRIENDS,

irst, the good news: A team led by Dr. Stephan Züchner at the
FUniversity of Miami has discovered a new type of CMT that may be
treatable with drugs already approved for diabetes. If those drugs show
promise, it may be possible to accelerate the clinical trial timeline and
skip straight to Phase 2 since the drugs have already gone through
Phase 1 testing for other indications.
Researchers think that at least 3,000–5,000 people in the United
States—and more than 60,000 worldwide—have this type of CMT,
making it the most common recessive form of the disease. “We’re very optimistic about the
therapy going forward,” Dr. Züchner said, noting that the drug basically cured CMT in
fruit flies. “This is a remarkable discovery only possible due to large scale genomic data
aggregation from volunteering CMT patients,” he added.

There is no bad news, but because we’re all still dealing with the pandemic, we are contin-
uing to seek out and bring you helpful information for dealing with it. Resident psychotherapist
David Tannenbaum offers advice on getting a handle on fear, the first step to a life with much
less worry.
Dr. Mario Saporta, director of the Center of Excellence at the University of Miami, shares
what happens when CMT patients turn to telemedicine, and Dr. Ashraf Elsayegh answers the
question: Are CMT patients at increased risk from intubation and mechanical ventilation?
Since COVID-19 has forced us to move so many CMTA community services online, we
asked Laurel Richardson, our director of community outreach, to share what’s happening
online, everything from chair dancing lessons on Facebook Live, to virtual walks, Zoom support
meetings and much, much more.
Finally, because it is summer, albeit a strange one, we bring you two stories that speak to
the essence of the season—one by an author who found the courage to wear shorts at the ripe
old age of 28 and the other about a group of kids who created a classic lemonade stand to
raise money for CMT research.

All of us at the CMTA are staying active, staying committed, staying hopeful and staying
focused on our main goal—the eradication of CMT. We hope you’re doing the same.
All my best,

AMY GRAY, Chief Executive Officer

SUMMER 2020 THE CMTA REPORT 3