Small Molecule Therapies for CMT
Small molecule therapies typically are developed from simple building blocks or are derived from natural sources. With their small size, they are easily able to pass through the cell membrane and access targets inside the cell.
Small molecule drugs are designed to modify a disease process via regulation of a biological target such as an enzyme, channel or receptor. They make up the majority of today’s drug treatments and may be used alone or as individual elements in combination therapies.
CMT Research Foundation is leading the charge to ensure safe and effective therapies for all forms of CMT make it to the market to help those living with CMT today. We are working every day to support the families, the foundations, the academics, the scientists, the biotechs and the pharma companies who share our goal and who are working to make it happen.
Related News & Research
Living in Pain Every Day is Not Rare to Me
The day after my 29th birthday, I received confirmation that the intense pain I’d been experiencing all my life was most certainly not in my head. It was Charcot-Marie-Tooth 1X. During the first year after my diagnosis, I fell into a deep depression. My family had a hard time accepting this diagnosis. Telling my story has changed my life, and I’m determined to change the future. Read more.
Worrying About My Daughter’s Future is Not Rare to Me
CMT may be a rare disease, but it is anything but rare to me. The most challenging part of living with CMT is the emotional toll it takes. I often think about the significant moments of my life that are yet to come: Will I be able to take my daughter to a father-daughter dance? Will I still be able to walk her down the aisle when she gets married? Read more.
Additional Funding Announced for Promising Gene Silencing Approach to Treat CMT1A
After demonstrating tremendous progress, the CMT Research Foundation’s Scientific Advisory Board has decided to extend funding for a research project led by Dr. Kleopas Kleopa for six more months. If successful, this project could lead to the first genetic therapy for CMT1A. Read more.
Caring for Someone with CMT: 3 Tips for CMT Caregivers
With no treatments or cures currently available for people with Charcot-Marie-Tooth disease, caring for someone with CMT may feel daunting. As a dad of a daughter with CMT and a friend to many with the disease, I know first-hand how physically and mentally challenging living with the disease can be. Here are three ways to help someone with Charcot-Marie-Tooth and be a supportive CMT caregiver.
CMT Research Foundation Launches Groundbreaking Research to Overcome Barriers to Delivering CMT Therapies to the Peripheral Nervous System
The CMT Research Foundation has launched a groundbreaking new research collaboration today with James Dahlman, Ph.D., assistant professor in the Wallace H. Coulter Department of Biomedical Engineering at the Georgia Institute of Technology and Emory School of Medicine. This innovative partnership seeks to overcome the challenges in CMT drug delivery by using lipid nanoparticles (LNPs) to deliver therapies to the PNS that could slow, stop or even reverse the effects of CMT in patients.
What to Expect in CMT Research During 2021: 5 Questions with Chief Scientific Officer Keith Fargo
What progress can patients and families expect to see in CMT research during 2021? What is the most promising research on the horizon? The CMT Research Foundation’s Chief Scientific Officer Keith Fargo, Ph.D., sat down with us to answer your most pressing questions about CMT research in the year ahead.
Dr. Bruce D. Carter Joins Scientific Advisory Board to Help CMT Research Foundation Deliver Treatments and Cures
Bruce D. Carter, Ph.D., has joined the CMT Research Foundation’s Scientific Advisory Board to help guide research strategy and funding to deliver treatments and cures for CMT. Dr. Carter is a professor of biochemistry and the associate director of the Brain Institute at Vanderbilt University. Read more about why he’s passionate about ending CMT and how he’ll be helping the CMT Research Foundation in its quest to do that.
Peter J. deSilva Joins CMT Research Foundation Board of Directors to Help Speed Treatments & Cures
The CMT Research Foundation is pleased to welcome Peter J. deSilva as the newest member of our board of directors. With more than 30 years of experience as a results-oriented leader in the financial sector, Mr. deSilva will bring his world-class expertise in strategic development and financial management to help the CMT Research Foundation raise significant funds for cutting-edge research and scientific innovation. Read more.
Progress is Possible Because of You
As we begin 2021, I am overwhelmed with gratitude for your continued commitment to the CMT Research Foundation community. Despite a challenging last year, friends like you rose to the challenge and have made it possible for the CMT Research Foundation to fund new and...
How to Measure Progress in CMT Research: 8 Signs Research is Working
It costs more than $2.6 billion to develop an approved prescription medicine and typically takes between 10 to 15 years to get a drug to clinical trials. With no treatments or cures currently available for diseases like CMT, it’s easy to question how donations to support scientific research make a difference. These 8 signs let you know when research is working.
