April 2013

CMT research consortium looks for a cure

The Charcot-Marie-Tooth Association (CMTA) is continuing progress with its STAR (Strategy to Accelerate Research) Initiative, which it created to accelerate CMT research.

The STAR initiative, which has recently reached several research milestones, provides a structure for collaboration among an international consortium of peripheral neuropathy researchers and clinicians who are leveraging the identification of 51 gene defects involved in CMT to develop treatments and cures for the disease, which affects 2.6 million people worldwide.

Earlier this year, the CMTA announced the launch of STAR Pathways, the integration of a directed protocol to determine effective CMT treatments.

Consortium scientists have already completed some major phases of the protocol—creating cell lines and an animal model for testing and screening cell lines at the National Institutes of Health to diagnose impact on the disease.

Next steps are preclinical study of compounds that regulate CMT, investigating safety of new medications, and two phases of human trials.

Over the next two years, the CMTA plans to raise $25 million to sponsor research targeting type 1A CMT, the most common form of the disease. The research protocol is also being used to study types 2A and 2E.

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