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
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...
CMT Research Foundation and Nanite Inc. Announce Partnership to Enhance Therapeutic Efficacy of Antisense Oligonucleotides in CMT1A
ATLANTA, GA (February 22, 2023) The CMT Research Foundation (CMTRF), a non-profit focused solely on delivering treatments and cures for Charcot-Marie-Tooth (CMT) disease*, today announced a partnership with Nanite, Inc., a Boston-based biotechnology company developing...
‘Amazon Smile’ Program Discontinued
On February 20th, 2023, Amazon ended their Amazon Smile Charity Program. Thank you to the nearly 200 people who supported the CMT Research Foundation through this program. In total, the community was able to raise:Interested in finding a new way to make a difference,...
Continuation of Inflammation Research for CMT1B
In 2020, the CMT Research Foundation had funded renowned nerve disease expert, Dr. Rudolf Martini of University Hospital Würzburg (Germany), to investigate whether inhibiting inflammation in the peripheral nerves could reduce symptoms and improve outcomes for patients...
Optimal Dosing Identified in CMT1X Research Update
A CMT Research Foundation-funded project at the University of Illinois Chicago has identified optimal dosing for a potential therapeutic for CMT1X. Like other forms of CMT, cell-to-cell communication within the peripheral nervous system malfunctions in people with...
CMT2E Gene Therapy Project Successfully Completes Milestone 2
A collaboration between Dr. Chris Lorson and Dr. Michael Garcia of the University of Missouri and funded by the CMT Research Foundation is producing promising results in an innovative new approach to treating CMT2E. CMT2E presents a special challenge in that the...
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