Columbia receives up to $15 million from Spinal Muscular Atrophy Foundation

the second most common autosomal recessive disease a disease caused by a mutation of a single gene passed on by both parents SMA has not received as much attention as many other less-common genetic disorders.

Since its inception, the SMA Foundation, led by co-founders Dinakar Singh and Loren Eng, has pledged nearly $30 million to researchers at academic medical centers and biotech companies. The grant to the Center for Motor Neuron Biology and Disease is meant to unite, consolidate and amplify previous funding for SMA research and clinical efforts at Columbia University. These include:

The SMA Clinic at Columbia University Medical Center, directed by Darryl De Vivo, M.D., the Sidney Carter Professor of Neurology The Pediatric Neuromuscular Clinical Research Network, a multi-center network that will conduct clinical trials in SMA patients as drug candidates are discovered. The center is also led by Dr. De Vivo.

A 2004 Young Investigator Award in Spinal Muscular Atrophy to Umrao Monani, Ph.D., assistant professor of neurology, who was instrumental in developing a mouse model of SMA Brent Stockwell, Ph.D., assistant professor of biological sciences and chemistry, who uses new technology to screen tens of thousands of compounds for potential SMA drugs. In 2004, indoprofen, a close cousin of ibuprofen, was the first candidate identified by this new screening technology.

About Spinal Muscular Atrophy
Spinal muscular atrophy is a genetic, motor neuron disease characterized by the wasting of skeletal muscles due to progressive degeneration of nerve cells in the spinal cord. Loss of function leads to premature death due to respiratory problems; half of its victims die before reaching age two. The SMA Foundation estimates that there are currently 25,000 to 55,000 people suffering from SMA in the United States, Europe, and Japan and that the annual market potential for a drug to treat SMA could exceed $500 million. '"/>

Contact: Elizabeth Streich
Columbia University Medical Center

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