News & Stories

See the latest news about CMT drug development and read stories from the CMT community that highlight why we must deliver treatments and cures during our lifetime.

CMT Research Foundation Announces New Research to Study Inflammation as Potential Treatment Target for Charcot-Marie-Tooth Disease

Oct 6, 2020 | CMT Research Updates, CMTRF Funded Research, Drug Development, Research News

The CMT Research Foundation is proud to announce a new research collaboration with renowned nerve disease expert Dr. Rudolf Martini of University Hospital Würzburg to investigate whether inhibiting inflammation in the peripheral nerves could reduce symptoms and improve outcomes for patients with Charcot-Marie-Tooth disease Type 1B. Read more about this project and see the four stages in the project.

WATCH: Power of the Patients: How We Change the Pace of Drug Development

Sep 24, 2020 | Drug Development, Life with CMT

The CMT Research Foundation is asking and answering the most pressing questions patients have about the need for treatments and cures. In episode 2, CMT Research Foundation CEO Susan Ruediger interviews Cure SMA’s Chief Scientific Officer Dr. Jill Jarecki, DTx Pharma Scientist Dr. Raffaella Gesuete and patient advocate Gary Donaldson to understand what patients can do today to influence the drug development process and speed progress. {HINT: Patients have the power to change everything!}

CMT Research Foundation Partner DTx Pharma Wins Grant from National Institutes of Health for CMT1A Gene Therapy Program

Sep 22, 2020 | CMTRF Funded Research, Drug Development, Research News

CMT Research Foundation partner DTx Pharma, Inc., has won a grant from the National Institutes of Health (NIH) to further fund its work on CMT. The investment is significant because the CMT Research Foundation gave DTx its first CMT-related grant just nine months ago....

WATCH: When Can Patients Expect Treatments for CMT?

Aug 31, 2020 | Drug Development, Life with CMT

In this first episode of the CMT Research Foundation’s new video series that asks and answers the toughest questions about CMT treatments, leading research experts will have you on the edge of your seat as they share when patients can expect treatments for CMT and how they can participate in accelerating progress.

5 Ways CMT Patients Can Take Action to Accelerate Drug Development

Aug 24, 2020 | Drug Development, Life with CMT

Unlike other neuromuscular diseases, CMT patients have no approved treatments. See how we’re applying breakthroughs in SMA to advance CMT drug development.

How Breakthroughs in Other Diseases Can Impact CMT

Jul 30, 2020 | CMTRF Funded Research, Drug Development

Unlike other neuromuscular diseases, CMT patients have no approved treatments. See how we’re applying breakthroughs in SMA to advance CMT drug development.

Nothing Mild About CMT

Jun 26, 2020 | CMT Research Updates, Drug Development

By: Susan Ruediger, CEO, CMT Research Foundation When I talk to individuals with CMT or share my personal experience about living with the disease, the responses I hear can be heartbreaking, sometimes even infuriating. “At least you don’t have cancer.” “You don’t look...

DTx Pharma Achieves Second Milestone in Study to Advance Treatments for CMT1A

Jun 26, 2020 | CMT Research Updates, CMTRF Funded Research, Drug Development, Research News

The CMT Research Foundation is pleased to announce that our partner DTx Pharma just completed the second milestone in a study we’re funding which uses their proprietary technology to suppress the overexpression of PMP22, the protein that causes CMT1A. Their work has...

Nothing Stops Innovation: Results from BIO 2020

Jun 24, 2020 | CMT Research Updates, Drug Development, Press Releases

By: Susan Ruediger, CEO, CMT Research Foundation “We’re not currently working in CMT, but we know we should be.” “We think our drug could work for CMT, but it has never been tested.” “CMT is definitely on our interest list for expansion, but we need to learn more...

CMT Genetic Testing: What’s Involved?

Jun 16, 2020 | About CMT

During the past several decades, researchers have discovered more than 100 genetic mutations that cause CMT. In the same time frame, scientists have developed tests that can look at parts of someone’s genetic makeup and identify changes that could cause certain...