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SMA Foundation and Rules-Based Medicine Collaborate on Biomarker Panels to Guide SMA Therapeutic Development

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In this collaboration, RBM will discover and also confirm plasma protein biomarker candidates previously identified from the multicenter Biomarkers for SMA (BforSMA) clinical study sponsored by the SMA Foundation and conducted by BG Medicine. The identification of such biomarkers may help to assess drug efficacy and shorten the duration of clinical trials of SMA therapies.

In this initial stage of the collaboration, the SMA Foundation provided BforSMA plasma samples to RBM for processing on the DiscoveryMAP™ platform plus an additional set of 70 biomarker assays.  Candidate biomarkers were found that significantly differentiated between disease and control groups and correlated with SMA disease severity.  The SMA Foundation and RBM are continuing to analyze these results and plan to create a specific panel of biomarker assays for use in clinical trials exploring new treatments for SMA.

"Biomarkers are critical to accelerating therapeutic development for any disease. They are even more important for rare diseases like SMA where patient populations are small and mostly consist of children," said Karen Chen, Ph.D., Chief Scientific Officer of the SMA Foundation. "We are excited to work with RBM to leverage their MAP technology to develop a biomarker panel for SMA that will help evaluate responses to treatments more efficiently and minimize the burden of clinical trials for patients and families."

"We are delighted to support the efforts of the SMA Foundation to develop better treatments for this tragic disease," said Craig Benson, Chief Executive Officer of RBM.  "Our MAP technology has been effective as part of many therapeutic development programs and for identifying biomarker patterns linked to complex diseases.  The confirmation and validation of biomarker patterns on our platform and the availability of testing services through our CLIA-certified lab will accelerate the development of new treatments for SMA."