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 Partners with Jenny Decker, CMT Patient Attempting to Solo-Circumnavigate Globe to Raise Awareness of Charcot-Marie-Tooth Disease
The CMT Research Foundation (CMTRF) a non-profit focused solely on delivering treatments and cures for Charcot-Marie-Tooth disease (CMT)*, has partnered with Jenny Decker, a 40-year-old nurse battling CMT, a rare neurological disease with no known cure, who last...
CMT Research Update/NMD Pharma
NMD Pharma recently published data demonstrating that their drug candidate, NMD670, improves muscle function and neuromuscular transmission deficits in both animal models and patients. Dr. William David Arnold, a member of the CMTRF Scientific Advisory Board, who was...
Vanderbilt’s Charles Sanders Successfully Finds Molecules That Alter PMP22 Production or Cell Surface Trafficking; In Next Phase Will Test if They Can Improve CMT-like Problems in Schwann Cells
CMT1A is caused by a gene-copying event that results in the overproduction of the peripheral myelin protein 22 (PMP22) in Schwann cells. The excess PMP22 protein fails to traffic normally to the cell surface and instead collects inside the cells as clumps of proteins...
CMT Research Foundation Partners to Advance Study of CMT1J by Dr. Stephan Zuchner
The CMT Research Foundation, a non-profit focused solely on delivering treatments and cures for Charcot-Marie-Tooth disease (CMT), has partnered with the 1J Foundation, a 501(c)(3) organization dedicated to finding a cure for patients with 1J, a newly identified...
Project Update: Nanite Looks to Advance Polymer-Based Encapsulation for CMT1A
CMT 1A results from the duplication of the myelin protein 22 gene (PMP22) in Schwann cells. This causes excessive production of the PMP22 protein which disrupts the myelination process of peripheral nerves and ultimately results in axonal loss and muscle wasting over...
Cisco’s Santo Caivano Joins CMT Research Foundation Board of Directors
CMT Research Foundation (CMTRF), a patient-led, non-profit focused on delivering treatments and cures for Charcot-Marie-Tooth (CMT) disease, today announced that Santo Caivano, Director, Customer Engagements of Cisco’s CAP (Critical Accounts Program) Americas team,...
CMT Research Update/Armatus Bio
Armatus Bio has partnered with Andelyn Biosciences, Inc., a cell and gene therapy Contract Development and Manufacturing Organization to accelerate manufacturing of an Armatus gene therapy treatment for Charcot-Marie-Tooth Type 1A. The goal is to maximize program...
Kayleena Speakman Joins The CMT Research Foundation as Communications Manager
Kayleena Speakman has joined The CMT Research Foundation, a patient-led, non-profit focused on delivering treatments and cures for CMT as Communications Manager responsible for emails, blogs, social media posts and opinion pieces for the CMT community, donors,...
An Extraordinary Year and the Road Ahead
As we begin 2024, I want to take a look back and celebrate the extraordinary year we had at the CMT Research Foundation. Since 2018, CMTRF has had an outsized impact on inspiring drug hunters to work on developing therapeutics for CMT and establishing itself as a key...
Updates on CMT Research
There have been a number of interesting developments in CMT research in the past few weeks. Here are three: Update 1: Pharnext recently announced topline results of its pivotal phase 3 (PREMIER trial) of PXT3003, its drug candidate for Charcot-Marie-Tooth 1A (CMT1A)...