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

Hope Alone Won’t Save My Hands, But a Treatment Will

by | Nov 23, 2020 | ,

I heard a loud pop. Then came the sudden swelling. All I’d been doing was walking down the sidewalk when my left leg snapped. At first, I thought it was just sprained. I kept limping on it for two weeks, until a doctor confirmed it was broken. Doctors gave me three options: Live with the pain and wear a removable cast for the rest of my life, undergo another surgery that probably won’t work, or amputate a portion of my leg. I chose amputation. Read more about Joe’s journey and what he’s doing to give people with CMT more than hope.

Thank You for Giving Me More Than Hope

by | Nov 19, 2020 | ,

This year has brought great momentum in the fight against CMT. At the CMT Research Foundation, we are hearing from scientists that it feels like something special is in the air. Conferences and journals are full of new discoveries, fundamental problems in CMT are being solved, new genes are being discovered, and new drugs are being developed. Read more about the progress.

How We Get from Today to Approved CMT Treatments: An Interview with FDA Director Dr. Peter Marks

by | Oct 29, 2020 | , ,

The CMT Research Foundation is asking and answering the most pressing questions patients have about the need for treatments and cures. In episode 3, CMT Research Foundation CEO Susan Ruediger interviews the FDA’s Dr. Peter Marks to discuss gene therapies as a potential treatment option for CMT, what the approval process looks like and how patients can expedite it.

Curcumin and CMT: What You Need to Know

by | Oct 27, 2020 | , ,

“Should I be taking curcumin supplements or eating more turmeric?” Many people with Charcot-Marie-Tooth disease (CMT) have been asking this question after researchers recently published a paper about curcumin and CMT in the journal Free Radical Biology and Medicine. The CMT Research Foundation’s chief scientific officer explores the findings and what it means for people with CMT.

CMT Research Foundation Launches New Research Project to Design Precision Medicine Approach for Charcot-Marie-Tooth Disease

by | Oct 14, 2020 | , , ,

In this new CMT Research Foundation-funded project, Drs. Lorson and Garcia at the University of Missouri will develop and test a new gene therapy approach that will both silence the abnormal gene and simultaneously replace it with genetic material that will produce normal protein. The research team will test this approach in an animal model of CMT2E that is already well understood, making it suitable for this proof-of-concept project. If the approach is successful, it could potentially be used for other forms of CMT as well. In fact, it is possible this approach could be used for many CMT-causing mutations that require both silencing of a mutated gene and replacement with normal protein — whether the mutation is currently known or has yet to be discovered. The ability to silence and replace genes, regardless of the specific mutation, is what makes this a precision medicine approach.

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