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
DTx Pharma
We are pleased share this story that published on May 3: https://patientworthy.com/2023/05/03/dtx-pharma-developing-cmt1a-rna-therapeutics/ One of the early programs CMTRF funded was DTx, a young company that had developed a new platform for RNA therapeutics. DTx was...
DTx Pharma Video – Using siRNA to Treat Charcot-Marie-Tooth Disease Type 1A
Thomas F. Sander Joins CMT Research Foundation Board of Directors
ATLANTA (April 26, 2023) CMT Research Foundation (CMTRF), a patient-led, non-profit focused on delivering treatments and cures for Charcot-Marie-Tooth disease, today announced that rare disease sales specialist, Thomas F. Sander, has joined its Board of Directors....
Edritz Javelosa to Join the CMT Research Foundation as Chief Scientific Officer
ATLANTA (April 17, 2023) Edritz Javelosa (Hav-a-low-sa), PhD, who has been at the Muscular Dystrophy Association (MDA) since 2019 and Research Portfolio Director since 2021, has joined the CMT Research Foundation (CMTRF) as Chief Scientific Officer. Reporting to...
The CMT Research Foundation Appoints Anna Combes Vice President of Philanthropy
ATLANTA (April 10, 2023) Anna Combes has joined the CMT Research Foundation in the newly-created position of Vice President of Philanthropy. Reporting to the Chief Operating Officer, Ms. Combes will be responsible for fundraising and development activities, including...
The CMT Research Foundation (CMTRF) Joins the European CMT Federation (ECMTF)
The CMTRF is happy to announce that they are joining the ECMTF, a non-profit organization grouping European and worldwide national associations supporting people affected by the Charcot-Marie-Tooth (CMT) disease or neuromuscular disorders. One of the primary goals of...
Peter de Silva, Board Member of the CMT Research Foundation and Former Retail President of TD Ameritrade, Publishes Taking Stock
Taking Stock engagingly toggles back and forth between de Silva’s highly successful business career and his quiet battle with CMT ATLANTA (March 23, 2023) Peter de Silva, a nationally recognized and highly accomplished financial industry executive whose career...
CMT Research Foundation Invests in Vanderbilt University School of Medicine to Advance Research for CMT1A
ATLANTA (March 13, 2023) The CMT Research Foundation, a non-profit focused solely on delivering treatments and cures for Charcot-Marie-Tooth disease*, has invested in a project at Vanderbilt University School of Medicine Basic Sciences that seeks to treat the problem...
CMT Research Foundation Collaborates with Augustine Therapeutics on New Potential Therapy for Charcot-Marie-Tooth Disease
ATLANTA (March 2, 2023) The CMT Research Foundation (CMTRF), a non-profit focused solely on delivering treatments and cures for Charcot-Marie-Tooth disease (CMT)*, is pleased to announce a collaboration with Augustine Therapeutics, a Belgian pharmaceutical company...
A Rare Disease Throughout Time
More than 3 million people around the world have Charcot-Marie-Tooth (CMT) disease, but even with its quirky name, CMT is still relatively unknown. A progressive, degenerative disease involving the peripheral nerves that branch out from the brain and spinal cord to...