The CMTA is funding a record amount of research:
- STAR (Strategy to Accelerate Research) has identified compounds that down regulate PMP22 both in vitro and in vivo!
Two full time specialists at the NIH focused on High Throughput Screening and assay development.
Dr. Chan at CalTech focused on creating Type 2 cellular assays.
Dr. Zuchner at the University of Miami focused on creating a Type 2 rat model.
Dr. Svaren at the University of Wisconsin focused on Type 1 and Type 2 cellular assays.
Dr. Battacharrya at the University of Wisconsin focused on human IPS cellular assays.
Dr. Klaus Nave and Dr. Michael Serada at the Max-Planck Institute, Germany, focused on testing compounds in vivo Type 1.
Dr. Rudolf Martini, University of Wurzburg, Germany, focused on a stem cell pilot rat study for Type X.
Dr. Ron Liem, Columbia University, focused on creating cellular assays for Type 2E.
To complement the ongoing research, the CMTA has added Dr. Larry Wrabetz, University of Buffalo, a world renowned expert on CMT, and Dr. Mark Scheideler, Founder, HumanFirst Therapeutics, to the STAR team. Mark will help guide the CMTA's drug development path forward, helping to forge the necessary partnerships with the pharmaceutical industry in 2013!
Most importantly, the STAR process, namely replicating the various types of CMT in cellular assays suitable for High Throughput Screening of compounds, followed by animal model confirmation, ultimately leading to an approved treatment drug, has been proven valid by the Cystic Fibrosis Foundation. Their first FDA-approved drug for treatment of a devastating genetic disease went on the market this year and was discovered using a similar process to the one that the CMTA calls STAR!
Also, the CMTA surpassed last year's record year in fundraising!
Support and Action Groups have grown to 53 in 40 States, with Canada and Mexico now aboard!