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As multiple drug companies pursue treatments for FSHD, there is an urgent need to define the clinical trial strategies which will hasten drug development, including creating disease-relevant outcome measures and optimizing inclusion criteria. This proposal will develop two new outcome measures and optimize eligibility criteria by testing 160 patients in 7 sites over a period of 18 months.

Identification of several types of small noncoding RNAs including known microRNAs that are differentially expressed in FSHD2 muscle cells compared to control.