CMT Research Foundation Calls for Action During Awareness Month

Marta Figueiredo, PhD avatar

by Marta Figueiredo, PhD |

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CMT Awareness Month, action

The CMT Research Foundation (CMTRF) wants to turn September from Awareness Month into “CMT Action Month.”

This year, the foundation is seeking to turn awareness of Charcot-Marie-Tooth (CMT) disease into action through useful and informative videos, personal fundraising pages, and a “double-your-donation” fundraising campaign.

Launched in 2018 by two CMT patients, Susan Ruediger and Patrick Livney, CMTRF is a nonprofit group whose one mission is “to deliver treatments and cures to end CMT.”

The CMTRF is committed to funding the most promising and effective research focused on therapy development for CMT by establishing strategic alliances with academic and industrial partners.

While nearly 3 million people worldwide are affected by these inherited disorders, “to most of the world, CMT is a blank stare — something they’ve never thought about or even heard of,” Ruediger said in a press release. She was diagnosed with CMT1A when she was 18 months old and represents the fifth generation of CMT in her family.

After more than 130 years since its discovery, there are still no treatments or cures, the foundation notes.

CMT Awareness Month, observed every September, is a time to educate the world about CMT and raise awareness. That is key to improving the diagnosis, understanding, and management of this group of inherited peripheral nerve disorders, according to the foundation.

But “we need more than awareness. We need action,” Ruediger said.

Therefore, this year, the CMTRF invites everyone to “turn awareness into action,” renaming September as CMT Action Month.

“It’s easy to believe that a single action by one person is just a lone raindrop in a bucket,” Reudiger said. “But combined together, our individual droplets create a tidal wave that will forever change our future.”

One of the foundation’s new initiatives is the launch of a video series in which leading experts in research and therapy development answer often-avoided questions about CMT treatments (or lack thereof) and highlight what the patient community can do to accelerate the development of treatments and cures.

The first episode, which aims to demystify the therapy development process, is already available. Upcoming episodes will focus on how patients and patient-led organizations can speed up therapy development, and on clinical trial design and the therapy approval process at the U.S. Food and Drug Administration.

CMTRF also plans to release conversations with leading scientific experts in CMT to provide the latest updates of their projects and the progress patients and their families can expect.

The foundation also proposes two ways for people to take action to accelerate CMT research: one by donating and the other by starting a fundraiser.

The first, based on a “double-your-donation” system, seeks to raise monies for cutting-edge research projects that could go unfunded without additional support. Through Sept. 30, a generous group of donors will match every donation  — dollar for dollar — up to $30,000, meaning that every contribution will have twice the impact for CMT research.

The second initiative, part of the CMT Research Champions program, includes a digital platform that allows the creation of a personal fundraising page, which can be shared with family, friends, coworkers, and other networks.

Besides donating and starting a fundraiser, CMTFR highlighted four other ways that people with CMT can take action to speed up treatment development: visit a CMT clinical center, enroll in a CMT patient contact registry, help pharmaceuticals understand why they must invest in CMT now, and create awareness within friends and family.

Of note, CMT patient contact registries help recruit participants for clinical trials, while evaluating how the disease affects a person’s quality of life and how it progresses over time.

The Hereditary Neuropathy Foundation also is planning several virtual activities to raise awareness and research funds over this month. Its theme is “CMT is Ruff,” in appreciation of the service dogs on which many people with the disease rely.