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
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...
Italian Research Group Completes CMT Research Foundation-Funded Project, Results Encourage Further Work in New Therapy for Adult and Elderly Patients with CMT1A/CMT1B
The CMT Research Foundation is pleased to announce that Dr. Maurizio D'Antonio's research team at San Raffaele Scientific Institute in Milan, Italy has completed their CMT Research Foundation-funded project investigating the role of a protein called Gadd34 in a mouse...
2022 at CMTRF
Dear Friends of CMTRF, In preparing for another busy year at CMTRF, I took some time recently to look back at the accomplishments of 2022. I am immensely proud of what our team has achieved (summarized below) and I believe it underscores how CMTRF adds significant...
How to Answer: “What is CMT”
By Kenneth Raymond As a patient and an advocate and a student of the condition, I am often asked, “What exactly is CMT?”. My response is usually along the lines of: “CMT stands for Charcot-Marie-Tooth disease, a rare inheritable neuromuscular peripheral neuropathy...
Muscular Dystrophy Association and CMT Research Foundation Announce Joint Research Study Grant
(New York) December 14, 2022 The Muscular Dystrophy Association (MDA) and CMT Research Foundation (CMTRF) announce joint funding for a study by Alessandra Bolino, PhD, at San Raffaele Hospital (Ospedale San Raffaele), Italy, on restoring membrane trafficking in...
Dr. Arthur Suckow, DTx Pharma Inc. – FALCON
One of the first research teams the CMT Research Foundation (CMTRF) funded was DTx Pharma, Inc., and the project focused on delivery of genetic medicine to the myelinating Schwann cells of the peripheral nerves - a major scientific challenge to effectively treating...