Boston Neurobiologist Receives Grant to Battle Leading Genetic Killer of Infants and Toddlers



April 1, 2005 -- The Spinal Muscular Atrophy Foundation announced that the recipient of this year's Young Investigator Award in Spinal Muscular Atrophy (SMA) is Mustafa Sahin, MD, PhD, from Children's Hospital Boston. Dr. Sahin, a neurobiologist specializing in child neurology, will conduct new research on SMA - the leading genetic killer of infants and toddlers and a disease which has no known treatment or cure. The Young Investigator Award was established in 2003 as an incentive and mentoring program that allows a new generation of scientists the opportunity to initiate innovative research projects to accelerate the development of a cure for SMA.

Dr. Sahin's research project entitled, "The Role of SMN in Axon Outgrowth and Targeting" will examine the hypothesis that the protein SMN - produced by the SMN1 gene and is involved in the disease - may play a role in the transport or translational regulation of ribonucleic acids (RNA) necessary for axon growth or guidance in motor neurons. The grant provides Dr. Sahin with up to $375,000 for three years, which includes allowances for tuition, research expenses and an annual mentor stipend. Michael E. Greenberg, PhD, director of the Division of Neuroscience at Children's Hospital Boston and professor of neurology at Harvard Medical School, will serve as Dr. Sahin's mentor.    

"I am honored to receive this award and for the opportunity it allows me to apply my work on axon development to spinal muscular atrophy, a project that I am deeply committed to. Through better understanding about the cellular and molecular basis of spinal muscular atrophy, specifically the role of SMN protein in motor axons, significant progress can be made toward developing a treatment or cure for this fatal disease," said Dr. Sahin.

After receiving his bachelor's degree in biochemistry from Brown University and medical degree from Yale University, Dr. Sahin completed his internship and residency in pediatrics at The Children's Hospital of Philadelphia; followed by a residency in pediatric neurology at Children's Hospital Boston, where he now serves as an instructor of neurology. He has received numerous awards and honors including the Outstanding Junior Member Award by the Child Neurology Society.

"This grant provides critical support that will allow Dr. Sahin the opportunity to explore important new directions in SMA research," commented Dr. Greenberg. "We are very optimistic about expanding our research efforts and further increase our understanding of SMA so that we may find a treatment for this disease."

The award will be presented at a special ceremony held during the 57th Annual Meeting of The American Academy of Neurology in Miami Beach, Florida on April 13, 2005. The Young Investigator Award is co-sponsored by the American Academy of Neurology Foundation.

The Young Investigator Award is one element of the Foundation's aggressive agenda to fund research initiatives which will accelerate the development of a treatment or cure for SMA. At the end of 2004, the SMA Foundation had funded more than $15 million in groundbreaking research projects that span the spectrum of basic, translational clinical research.

Spinal muscular atrophy is a genetic motor neuron disease caused by the progressive degeneration of nerve cells in the spinal cord and brainstem, leading to muscle weakness, respiratory complications and premature death. Over 50 percent of patients with SMA die before the age of two. The SMA Foundation estimates that there are currently 55,000 people suffering from SMA in the United States, Europe and Japan.

About SMA Foundation
The SMA Foundation is a nonprofit organization founded in 2003 dedicated to finding a treatment or potential cure for spinal muscular atrophy (SMA). The Foundation provides funding for the full range of research from basic to clinical work conducted in academic laboratories as well as corporate therapeutics development. In addition, the Foundation is committed to raising awareness through education and increasing federal funding and support for the disease. For more information on the Spinal Muscular Atrophy Foundation, visit www.smafoundation.org or call (646) 253-7100.

About the AAN Foundation
The American Academy of Neurology Foundation works with the American Academy of Neurology to promote research in the neurosciences and to advance public understanding of the disorders of the brain and nervous system. For more information, visit www.neurofoundation.org.





Boston Neurobiologist Receives Grant to Battle Leading Genetic Killer of Infants and Toddlers