University of California Davis – David Pleasure, MD. is funded by HNF to develop a CMT 2A mouse model that will be distributed through Jackson Laboratories. In addition, Dr. Pleasure will be testing therapeutic strategies to treat CMT in collaboration with Burke Institute under the direction of Dr. Raj Ratan. These treatments will cover small molecules and gene therapy approaches.
Brett Langley Ph.D. and Diana Willis Ph.D., (Burke Medical Research Institute, Weill Medical College of Cornell University) are doing “Proof of Concept” studies on an in vivo mouse model with CMT 2A to come up with therapeutic approaches that have promise on other neuromuscular diseases.