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 Announces New Research to Study Inflammation as Potential Treatment Target for Charcot-Marie-Tooth Disease
The CMT Research Foundation is proud to announce a new research collaboration with renowned nerve disease expert Dr. Rudolf Martini of University Hospital Würzburg to investigate whether inhibiting inflammation in the peripheral nerves could reduce symptoms and improve outcomes for patients with Charcot-Marie-Tooth disease Type 1B. Read more about this project and see the four stages in the project.
WATCH: Power of the Patients: How We Change the Pace of Drug Development
The CMT Research Foundation is asking and answering the most pressing questions patients have about the need for treatments and cures. In episode 2, CMT Research Foundation CEO Susan Ruediger interviews Cure SMA’s Chief Scientific Officer Dr. Jill Jarecki, DTx Pharma Scientist Dr. Raffaella Gesuete and patient advocate Gary Donaldson to understand what patients can do today to influence the drug development process and speed progress. {HINT: Patients have the power to change everything!}
CMT Research Foundation Partner DTx Pharma Wins Grant from National Institutes of Health for CMT1A Gene Therapy Program
CMT Research Foundation partner DTx Pharma, Inc., has won a grant from the National Institutes of Health (NIH) to further fund its work on CMT. The investment is significant because the CMT Research Foundation gave DTx its first CMT-related grant just nine months ago....
Susan B. Canavari Joins CMT Research Foundation Board of Directors
Deeply Experienced Brand Marketer to Help Increase Awareness of CMT and Accelerate Research ATLANTA (September 21, 2020) The CMT Research Foundation, a nonprofit focused solely on delivering treatments and cures for Charcot-Marie-Tooth disease during our lifetime,...
A Family’s Fight to Change Their Daughter’s Future
Eight-year-old Leonor struggles to walk, sit up straight and hold a glass of water. Other kids make fun of her because she has to wear leg braces. When Leonor was diagnosed with CMT, doctors said she’d ben in a wheelchair for life. Her family refuses to accept that fate, and so do we. There’s always something we can do. Learn how you can take action to end CMT.
WATCH: When Can Patients Expect Treatments for CMT?
In this first episode of the CMT Research Foundation’s new video series that asks and answers the toughest questions about CMT treatments, leading research experts will have you on the edge of your seat as they share when patients can expect treatments for CMT and how they can participate in accelerating progress.
CMT Patients & Families Need More Than Awareness
It has been more than 130 years since CMT was first discovered, yet there are still no treatments or cures. This CMT Awareness Month, we need more than awareness. We need action. Here’s what we can do together to move the needle now.
5 Ways CMT Patients Can Take Action to Accelerate Drug Development
Unlike other neuromuscular diseases, CMT patients have no approved treatments. See how we’re applying breakthroughs in SMA to advance CMT drug development.
Videos that Help Explain CMT (and the Challenges to Ending it)
Why aren’t there treatments for CMT yet? How does the drug development process work and why does it take so long?
In this CMT Research Foundation video series, we’ve answered the most frequently asked questions about CMT treatments and the search for a cure. Watch as leading experts in research and drug development discuss the challenges of brining an effective therapy to market and what the patient community can do now to accelerate treatments and cures.
CMT Research Foundation Adds New Chief Operating Officer and Chief Scientific Officer
Meet the CMT Research Foundation’s new Chief Operating Officer Erich Fasnacht. See how he plans to increase the number and scale of CMT research projects.
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