RESEARCH OVERVIEW
The CMT4B3 Research Foundation is the only organization solely focused on finding a treatment or cure for this life-threatening disease.
We are committed to exploring faster, cost-effective, and collaborative scientific approaches. Our work isn’t just moving us closer to a cure for CMT4B3, but is also advancing treatments for other rare diseases, impacting many lives beyond just a few.
GENE REPLACEMENT THERAPY
Gene therapy works by fixing or replacing faulty genes that cause health problems. Scientists use special carriers, called vectors, to deliver healthy genes into the body’s cells. Viruses, modified to be harmless, often serve as these carriers. Once inside, the new genes can help cells work properly, offering hope for treating diseases like CMT4B3.
DRUG REPURPOSING
Drug repurposing involves testing existing medications to see if they can slow CMT4B3’s progression while we work on long-term solutions. Because these drugs are already developed, they can be fast-tracked into trials, offering a quicker path to potential treatment.
DISEASE MODELS
Studying CMT4B3 in animal models is essential for understanding the disease. Mice, which share many genetic similarities with humans, are often used for this. In addition to mice, we are developing cellular, zebrafish, and rat models to better understand CMT4B3 and find effective treatments.