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
Welcoming New Expert Advisors
By Keith Fargo, Chief Scientific Officer The CMT Research Foundation’s Scientific Advisory Board is composed of distinguished scientists and clinicians who provide independent expert perspectives as key volunteers. Their contributions take several forms. Most visibly,...
CMT Research Foundation Elects Board Member Cleary Simpson to Chief Executive Officer
By Patrick Livney, Board Chair and Co Founder As we move from being a startup to the leading foundation seeking to cure CMT, we can look back with great pride on our progress to date: We have raised almost $10 million in three years, funded 12 projects (with two in...
Michele and Kent Stahl and Family Give $1,000,000 to the CMT Research Foundation to Develop Treatments for CMT1A
Atlanta (January 4, 2021) Michele and Kent Stahl and their family today announce a $1 million donation to support the CMT Research Foundation’s $10 million ENDGAME: the Campaign to End CMT1A. Since the soft launch of ENDGAME in September, families and friends with...
Why Michele and Kent Stahl and their family made a $1,000,000 gift to CMT Research Foundation to develop treatments for CMT1A
Michele and Kent’s son, Austin, was diagnosed with CMT1A when he was 16 years old. Having difficulty with his balance and walking, it took years for him and his family to understand the cause. His mother Michele recounts, “we spent seven years searching for an answer....
2021 Year in Review
2021 has been a banner year for progress in CMT drug development, much of which has been possible because of your partnership with the CMT Research Foundation – thank you! Despite the challenges presented by a global pandemic, we can be proud of all that we were able...
Teams at Cyprus Institute of Neurology & Genetics and Nationwide Children’s Hospital Complete CMT Research Foundation-Funded Project, Resulting Therapeutic Now Moving into Pharmaceutical Development
Update, May 17, 2022: This work is now published in The Journal of Clinical Investigation. By Keith Fargo, Ph.D., Chief Scientific Officer, CMT Research FoundationThe CMT Research Foundation is pleased to announce that Dr. Kleopas Kleopa, a renowned neuromuscular...
CMT Research Foundation funds cutting edge research to deliver therapeutics to the peripheral nervous system
The CMT Research Foundation has awarded nearly $100,000 to support a 13-month project to test a new therapeutic delivery system that may be capable of crossing the blood-nerve barrier and releasing therapeutic payloads to the Schwann cells and axons of the peripheral...
Changing One Family’s Legacy
As with many families, I am but one leaf in a family tree colored by CMT1A across its many limbs and branches. I have watched successive generations struggle with the disease’s progressively debilitating physical symptoms and persistent emotional toll: grandparents,...
The CMT Research Foundation Kicks Off September CMT Action Month
Each September we join with other organizations to build awareness for Charcot-Marie-Tooth Disease. As you know, CMT is little-known and poorly understood outside of the patient community and we celebrate each and every effort to educate the medical community, the...
Researchers at Cyprus Institute of Neurology & Genetics Complete Third Milestone of CMT1A Gene Therapy Project
The CMT Research Foundation is pleased to announce that Dr. Kleopas A. Kleopa and his team at the Cyprus Institute of Neurology & Genetics, Nicosia, have completed the third milestone of their 24-month CMT1A gene therapy project. CMT1A is caused by duplication of...
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