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
Why FDA acceptance of gene therapy in SMA matters to CMT
Novartis's announcement about FDA filing acceptance could translate to CMT On Monday, Novartis, the parent company of AveXis, announced that a license application for their gene therapy approach to treat Spinal Muscle Atrophy (SMA) was accepted by the FDA. This is a...
Fontan Surgery for Heart Defects May Not Be Advisable for CMT1A Patients, Case Study Finds
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Where We Started, How Far We Have Come and Where We are Going
The CMT Research Foundation offers hope to those living with CMT that a treatment and cure are on their way!
$1,000,000 pledge to CMT research
CMTRF Receives $1 Million Research Grant from Grandview Steers Foundation The pace of research for CMT just got faster. Atlanta (November 8, 2018) The newly launched CMT Research Foundation (CMTRF), the only 501(c)(3) not-for-profit organization focused solely on...
Key CMT Researchers added to Scientific Advisory Board
Gene Therapy, Adult Stem Cell and Clinical Experts are added to the CMT Research Foundation’s Scientific Advisory Board
Charcot Marie Tooth (CMT) Experts Launch National Nonprofit to Fund Only Treatments and Cures
CMT Research Foundation to speed dollars to most promising research Atlanta (September 19, 2018) Two experts in Charcot-Marie-Tooth (CMT), who also have the condition, today launched the CMT Research Foundation (CMTRF) as the only 501(c)(3) not-for-profit organization...
