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
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.
Mom with CMT Loses Her Leg but Gains Strength for Her Daughter
My doctor told me it was the worst case of CMT he had ever seen. We reviewed my options: A fusion with no end to my pain, or amputation. Here’s what I chose and what I’m doing to change things for my daughter.
More Than Hope: An Action Plan to End CMT
As we approach the end of 2020 and look ahead to a new year, it’s natural to feel hopeful about what the future may hold — hope for an end to the global pandemic; hope for happiness and good health for our families; and hope for an end to Charcot-Marie-Tooth disease (CMT). The CMT Research Foundation shares these hopes, but we know delivering treatments and cures for CMT quickly requires more than hope. It requires vision, commitment, wisdom, a strong plan, and most importantly — action.
See a preview of the CMT Research Foundation’s 2021 action plan.
‘Tis the Season for Scientific Breakthroughs
During a year when so many things have been postponed, changed or cancelled, there is one thing that hasn’t stopped — the CMT Research Foundation’s efforts to end CMT. Research continues, even during the holidays, to find treatments and cures as quickly as possible. These researchers will be working this holiday season to uncover scientific breakthroughs. Here’s how several of our researchers will be celebrating by continuing their work for you.
20 Most Popular CMT Stories & Resources from 2020
2020 has been a year of information overload. To help the Charcot-Marie-Tooth (CMT) community navigate these staggering statistics, we’ve developed a round-up of the 20 most read articles and stories about CMT on cmtrf.org during 2020. You were most interested in three key areas: 1) stories from the CMT community, 2) the latest in research development and discovery and 3) understanding CMT and specifics about the disease.
After 10 Surgeries in 10 Years to Address Deformities from CMT, This Young Woman is Fighting Back
Surgical recovery takes up to 12 weeks and requires me to take a leave of absence from my job, lay with my toes above my nose, spend most of the day alone, and give up my independence. I’m unable to shower on my own, walk without crutches, and have become very socially isolated, especially during COVID-19 quarantine. Given that I’ve had so many surgeries in such a short period of time, it’s difficult to maintain steady employment, let alone a full-time career. All of that is on hold due to my CMT.
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