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 Launches New Research Project to Design Precision Medicine Approach for Charcot-Marie-Tooth Disease
In this new CMT Research Foundation-funded project, Drs. Lorson and Garcia at the University of Missouri will develop and test a new gene therapy approach that will both silence the abnormal gene and simultaneously replace it with genetic material that will produce normal protein. The research team will test this approach in an animal model of CMT2E that is already well understood, making it suitable for this proof-of-concept project. If the approach is successful, it could potentially be used for other forms of CMT as well. In fact, it is possible this approach could be used for many CMT-causing mutations that require both silencing of a mutated gene and replacement with normal protein — whether the mutation is currently known or has yet to be discovered. The ability to silence and replace genes, regardless of the specific mutation, is what makes this a precision medicine approach.
CMT Research Foundation Announces New Research to Study Inflammation as Potential Treatment Target for Charcot-Marie-Tooth Disease
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
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
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....
A Family’s Fight to Change Their Daughter’s Future
Eight-year-old Leonor struggles to walk, sit up straight and hold a glass of water. Other kids make fun of her because she has to wear leg braces. When Leonor was diagnosed with CMT, doctors said she’d ben in a wheelchair for life. Her family refuses to accept that fate, and so do we. There’s always something we can do. Learn how you can take action to end CMT.
WATCH: When Can Patients Expect Treatments for 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.
CMT Patients & Families Need More Than Awareness
It has been more than 130 years since CMT was first discovered, yet there are still no treatments or cures. This CMT Awareness Month, we need more than awareness. We need action. Here’s what we can do together to move the needle now.
5 Ways CMT Patients Can Take Action to Accelerate 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.
A rallying cry for the CMT community: If not us, then who? If not now, then when?
By: Gary Donaldson The New York Times recently published an article about individuals living with rare genetic diseases who have become advocates for their conditions. These courageous champions are shaping research to change the course of their disease, despite the...
Reflections on a CMT Research Supporter
By Patrick Livney, Chairman of the Board Recently, I was contacted by Barton, a son of Charles G. Bentzin, who informed me of the passing of his father. Sadness aside, his reaching out touched me on two fronts. First, telling me how Charles “enjoyed talking with you...
Important Research News Announcements
- Genomic Studies in Charcot-Marie-Tooth Disease July 31, 2024
- Charcot-Marie-tooth disease type 2A: An update on pathogenesis and therapeutic perspectives March 5, 2024
- Accelerate Clinical Trials in CMT (ACTCMT) Study March 1, 2024
- Creation of a Schwann Cell Gene Regulatory Network February 29, 2024
- Activation of XBP1s attenuates disease severity in models of proteotoxic Charcot-Marie-Tooth type 1B [Preprint] February 2, 2024
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