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
Gene Therapy for Axonal Neuropathies
There are efforts underway to develop gene therapies for several of the CMT types 2 and 4.
Milestone 2 Completed for CMT1A Project from Cyprus Institute of Neurology and Genetics
In February 2020, Dr. Kleopa completed milestone 2 of the 24-month project and the results continue to be encouraging. The goal during this 6-12 month period of the project was to further optimize the amount of PMP22 gene silencing in a mouse model of CMT1A. Dr....
Creating multiple opportunities for success in CMT 1A
By Grace Pavlath, PhD By now you’ve likely heard that CMTRF is funding three genetic-based projects focused on therapy development for CMT1A: DTx Pharma, Shift Pharma and Cyprus Institute of Neurology and Genetics. Funding multiple complementary approaches gives...
CMT Research Foundation Funds Shift Pharmaceuticals to Advance Search for CMT1A Treatment
Shift to Develop and Analyze Novel Series of Drugs Designed to Control Expression of PMP22 Gene The CMT Research Foundation (CMTRF), a nonprofit focused solely on delivering treatments and cures for Charcot-Marie-Tooth disease, today announced it has partnered with...
Importance of Biomarkers in CMT
Using Biomarkers to Measure Effective Treatment Options for CMT
Targeting Axon Degeneration – a treatment for all types of CMT?
Preventing axon degeneration could stop the progression of CMT. Read about the efforts by pharma to stop axon degeneration.
Are We Prepared for Clinical Trials in Charcot-Marie-Tooth?
A Review of Clinical Trial Readiness for CMT BY CMT RESEARCH FOUNDATION'S SCIENTIFIC ADVISORY BOARD MEMBER DR. ALEXANDER ROSSOR Read the full paper In this issue of Brain Research, Mike Shy, Mary Reilly and I summarize the current array of outcome measures for...
2019 – A Year of Closing the Gaps in CMT Drug Development
Because of you, the pace of drug development for CMT accelerated! As we close the books on 2019, I am excited to share the progress the CMT Research Foundation has made this year. Because of you, these advancements were possible. We have grown considerably in the...
Partnership with DTx Pharma to optimize antisense oligonucleotides as a gene therapy for CMT1A
ATLANTA (December 20, 2019) The CMT Research Foundation (CMTRF), a nonprofit focused solely on delivering treatments and cures for Charcot-Marie-Tooth, today announced it has partnered with DTx Pharma, an RNA medicines company breaking open new therapeutic areas for...
A Robust Therapeutic Pipeline for CMT 1A
The majority of people with CMT have the 1A subtype which is due to duplication of the peripheral myelin protein 22 (PMP22) gene leading to breakdown of the myelin that surrounds peripheral nerves. With time this loss of myelin causes nerve dysfunction and...
Address
4062 Peachtree Road
Suite A209
Atlanta, GA 30319
Phone Number
404.806.7180
Media Inquiries
© 2024 CMT Research Foundation | Privacy Policy