Spinal Muscular Atrophy Research Team Donates $50,000 to Cure SMA to Fund SMA Patient Report Research


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Buffalo, New York-based not-for-profit organization Spinal Muscular Atrophy Research Team (SMART) recently announced it awarded $50,000 to Cure SMA to help fund ongoing research for a cure for this debilitating disease.

Since its founding in 2009, SMART has selected promising scientists and institutions to receive research grants to further SMA research. Some of these grants have gone to pivotal work at Johns-Hopkins University, Northwestern University & University at Rochester.

Last month the organization celebrated its 7th SMART Celebration, which was successful in raising $150,000 for SMA research. The event took place at the First Niagara Center, the home of the Buffalo Sabres. Thanks to the SMART Celebration series and other fundraisers, the organization has been able to raise $750,000.

The recently awarded $50,000 research grant will fund Dr. Chat Heatwole’s work at the University of Rochester. It will focus on advancing a more reliable means for adult SMA patients to report on relevant clinical study findings, as the increasing number of SMA drug studies necessitates a more effective way of assessing treatment efficacy across all patient demographics and disease severity. This research grant forms part of a $220,000 total awarded to Cure SMA.

To learn more about SMART and their upcoming events, visit www.smarthope.com.

About Spinal Muscular Atrophy

SMA is a rare, devastating motor neuron disease and one of the leading genetic causes of pediatric mortality, occurring in approximately 1 in every 6,000 to 10,000 newborns. It is characterized by the degeneration of nerves controlling muscles and voluntary movement, resulting in muscle weakness, atrophy, paralysis and eventually death. SMA is the result of a mutation or deletion in a gene called survival of motor neuron 1 (SMN1) that results in low levels of the SMN protein. The disease has currently no approved treatment.

Dallas-based gene therapy platform company AveXis Inc., a leader in developing treatments for rare and life-threatening neurological genetic diseases, has announced the completion of a $65 million Class D common stock financing. The proceeds will be used to advance the company’s ongoing Spinal Muscular Atrophy (SMA) research and clinical programs and to expand key operational capabilities.