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The Spinal Muscular Atrophy Foundation Announces a Biomarker Panel to Guide SMA Therapeutic Development

NEW YORK, NY | April 3, 2012 – The Spinal Muscular Atrophy (SMA) Foundation announced today the launch of a biomarker assay panel for SMA using Myriad RBM’s Multi-Analyte Profiling (MAP) technology platform. The SMA-MAP panel is designed to evaluate the severity of SMA and disease progression and can be used to assess drug efficacy and shorten the duration of clinical trials for SMA therapeutics.

The SMA Foundation initially provided more than 120 plasma samples from SMA patients collected in the Biomarkers for SMA (BforSMA) clinical study for processing on Myriad RBM’s DiscoveryMAP® 250+ biomarker discovery platform.  Analysis of this data revealed a set of candidate biomarkers which were then tested against over 150 longitudinal samples from an SMA natural history study by the Pediatric Neuromuscular Clinical Research Network.  A panel of 27 validated biomarkers was selected, and is now being made available to clinical researchers by Myriad RBM as a new product to be called SMA-MAP.   Measuring these biomarkers in SMA clinical trials may help researchers to efficiently predict and monitor SMA progression and therapeutic efficacy.

“Myriad RBM has given us a tool that holds great promise.” said Karen S. Chen, Ph.D., Chief Scientific Officer and Chief Operating Officer of the SMA Foundation. “We are now able to make biochemical measurements that will help us objectively assess functional changes in children and adults with SMA, and, hopefully, detect early signals of therapeutic success in new drug trials for this devastating disease.”

“Research on orphan diseases like SMA is typically underfunded.  As a result, the development of effective therapies and treatments proceeds slowly and is insufficient to address the critical need for children and families affected by medically devastating illnesses like SMA,” said Craig Benson, President of Myriad RBM. “SMA researchers now have access to a quantitative and reliable tool that links biomarker results with complex disease symptoms and characteristics.  We are confident that the use of the SMA-MAP will play an important role in accelerating development of SMA therapeutics.”

ABOUT SPINAL MUSCULAR ATROPHY

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 with SMA.

ABOUT THE SPINAL MUSCULAR ATROPHY FOUNDATION

Founded in 2003, the Spinal Muscular Atrophy Foundation is a nonprofit organization dedicated to accelerating progress towards a treatment for Spinal Muscular Atrophy through targeted funding of clinical research and novel drug development efforts. Since its inception, the Foundation has awarded over $100 million for SMA research. 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, visit the SMA Foundation website at www.smafoundation.org.

Contact:

SMA Foundation
Dione Kobayashi
dkobayashi@smafoundation.org
646-253-7100