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

NEW YORK and AUSTIN, Texas, Jan. 19, 2011 /PRNewswire/ -- The Spinal Muscular Atrophy (SMA) Foundation and Rules-Based Medicine, Inc. (RBM) announced today that they have reached the first milestone in a program to develop a panel of plasma protein biomarkers for SMA using RBM's Multi-Analyte Profiling (MAP) technology platform.  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."

About Rules-Based Medicine (RBM)

Rules-Based Medicine's biomarker testing service provides clinical researchers, physicians and healthcare providers with reproducible, quantitative, multiplexed data for hundreds of proteins to advance drug development and patient care. The Company's proprietary Multi Analyte Profiling (MAP) technology offers pre-clinical and clinical researchers broad, cost-effective protein analyses in multiple species from a small sample volume. MAP technology also supports RBM's drive to develop diagnostics that aid in the detection of complex diseases and conditions in areas of unmet medical need such as neuropsychiatry, nephrology, immunology and cardiology. RBM's initial molecular diagnostic test, VeriPsych™, is a blood-based test that aids in the confirmation of diagnosis of recent-onset schizophrenia by evaluating a proprietary set of 51 biomarkers. RBM also offers innovative and proprietary ex vivo testing systems such as TruCulture™, the first fully-closed, reproducible whole blood culture system. More information about RBM is located at


Spinal Muscular Atrophy (SMA) is a motor neuron disease and the leading genetic cause of death among infants and toddlers.  Characterized by selective loss of nerve cells in the spinal cord, the disease leads to increasing muscular weakness and atrophy. Patients afflicted by SMA lose muscle control and strength, leading to progressive inability to walk, stand, sit up and breathe, depending on the severity of the disease. It is estimated that approximately 1 in 6,000 -10,000 infants are born annually worldwide with SMA.


Founded in 2003, the Spinal Muscular Atrophy Foundation is a nonprofit organization dedicated to accelerating progress towards a treatment and cure for spinal muscular atrophy through targeted funding of clinical research and novel drug development efforts.  Since its inception, the Foundation has awarded over $60 million in sponsored research agreements.  In addition, the Foundation is committed to raising awareness and generating support for increased research efforts in SMA among the leaders of industry and government.  For more information on the Spinal Muscular Atrophy Foundation, visit or call (646) 253-7100.

SOURCE Spinal Muscular Atrophy Foundation; Rules-Based Medicine, Inc.
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