The Scientific Advisory Board was established in 1986. Through the Board's invaluable expertise, research projects at Families of SMA are evaluated. The members of the SAB annually review all applications for research grants, using a procedure similar to that used at NIH. Since 2004, the FSMA Scientific Advisory Board has awarded 66 grants for a total of $8,195,096 in funding.  Please click here to see a list of funded grants.  This group also helps plan the program of the Annual International SMA Research Group Meeting . We would like to thank the members of the Scientific Advisory Board for their expertise in ensuring that the "best" SMA research is funded by FSMA.

Jill Jarecki, Ph.D., FSMA Research Director.
Dr. Jarecki serves as the coordinator of the FSMA Scientific Advisory Board. Prior to joining FSMA, Dr. Jarecki studied neuromuscular development in graduate school at Yale University and was a post-doctoral fellow at Stanford University. Most recently she was a senior scientist at Invitrogen Corporation and also Vertex Pharmaceuticals, where she led a drug discovery program to identify potential small molecule therapeutics for SMA.

Current Members of the FSMA Scientific Advisory Board

Arthur H. Burghes, Ph.D.
Dr. Burghes is a Professor of Molecular and Cellular Biochemistry at the Ohio State University and an expert in the field of SMA biology.  His laboratory focuses on the molecular understanding of genetic neuromuscular disorders, in particular SMA. Dr. Burghes developed the first animal model of SMA and demonstrated that high copy numbers of the SMN2 gene can rescue the SMA mouse.  Dr. Burghes joined the SAB in 2002.  Please click here to see his web page at OSU.  

Tom Crawford, M.D.
Dr. Crawford is an Associate Professor of Neurology and Pediatrics at the Johns Hopkins School of Medicine.  He is co-director of the MDA clinic for Neuromuscular Disorders.  His practice involves general child neurology with a principal interest in caring for children with neuromuscular, neuromotor, and ataxic disorders. His primary research interests involve the basic science and clinical characterization of two important neurologic disorders that affect children: SMA and Ataxia Telangiectasia. He joined the SAB in 2002.  Please click here to see his web page at Johns Hopkins University.

Douglas Kerr, M.D., Ph.D.
Dr. Kerr is Director, Experimental Neurology at Biogen Idec, where he oversees clinical trials for small molecule drug development in ALS.  Dr. Kerr was formerly the Director of the Johns Hopkins Transverse Myelitis Center.  He has investigated neural stem cells as a potential tool for functional recovery in patients with motor neuron disease, such as SMA.  Kerr joined the SAB in 2005.  Please click here to see the Biogen Idec website.

Adrian Krainer, Ph.D.
Dr. Krainer is a Professor at the Cold Spring Harbor Laboratory.  His research interests include unraveling the mechanisms controlling pre-mRNA splicing, including in genetic diseases such as SMA.  He is a leading expert in this area with over 100 research articles published to date.  Dr. Krainer joined the SAB in 2005.  Please click here to see his web site at Cold Spring Harbor Lab.

Rashmi Kothary, Ph.D.
Dr. Kothary is an Associate Director and Senior Scientist at the Ottawa Hospital Research Institute and a Professor at the University of Ottawa. Dr. Kothary works on understanding the importance of the cytoskeleton in neuromuscular disorders such as SMA. Dr. Kothary has a keen interest in modeling disease pathology in mice and has developed an intermediate mouse model of SMA.  He also holds the University Health Research Chair in Neuromuscular Disorders. Dr. Kothary joined the SAB in 2010.  Please click here to see his website at Ottawa Hospital Research Institute.

Samuel Pfaff, Ph.D.
Dr Pfaff is an Investigator of the Howard Hughes Medical Institute and Professor at the Gene Expression Laboratory at the Salk Institute for Biological Studies in La Jolla, California.  The Pfaff laboratory focuses on the development of motor neurons.   Of special interest to him is how motor neurons develop and make connections between the spinal cord and muscles in the body, and how these connections relate to spinal cord injuries and to degenerative diseases such as ALS (Lou Gehrig's disease), spinal muscle atrophy and post-polio syndrome. In 2009, his group at the Salk Institute was awarded a $11.5 million grant by the California Institute for Regenerative Medicine (CIRM) for translational research focusing on developing a novel stem-cell based therapy for Amyotrophic Lateral Sclerosis (ALS). Dr. Pfaff joined the SAB in 2011. Please click here to see his website at the Salk Institute. 

Mark Rich, M.D., Ph.D.
Dr. Rich is an Associate Professor in the Department of Neuroscience, Cell Biology, and Physiology at Wright State University.  He completed a medical fellowship in neuromuscular disease and spends about 10% of his time with neuromuscular patients in clinic.  His research laboratory specializes on synapse physiology at the neuromuscular junction in mice.  Dr. Rich joined the SAB in 2010.  Please click here to his website at Wright State University.

Kathryn Swoboda, M.D.
Dr. Swoboda is an Associate Professor of Neurology and Pediatrics at the University of Utah School of Medicine, and Director of the Pediatric Motor Disorders Clinic at Primary Children’s Medical Center in Salt Lake City, Utah. The primary focus of her work is to better understand the pathophysiology contributing to muscle weakness in children with SMA and to help facilitate the rapid translation of new therapies for treatment trials.  She joined the SAB in 2002. Please click here to see her website at the University of Utah. 

Recently Retired Members of the FSMA Scientific Advisory Board

Families of SMA thanks each of the following individuals who recently retired from our SAB, after at least five years of service.  We very much appreciate the time, expertise, and dedication each donated to FSMA and our mission. 

Mark Gurney, Ph.D., M.B.A.
Dr. Gurney is Senior Vice President, Drug Discovery and Development at deCODE genetics, Inc. He was formerly Director of Genomics Research at Pharmacia Corporation. Prior to his position at Pharmacia, Dr. Gurney held academic appointments in the Department of Pharmacological and Physiological Sciences at the University of Chicago and in the Department of Cell, Molecular, and Structural Biology at the Northwestern University Medical School.  He has had a long and continuing interest in neurodegenerative diseases, having developed one of the first animal models of motor neuron disease. He joined the SAB in 2003.  Please click here to see his biography on the deCODE Genetics webpage.

Louise Simard, Ph.D.
Dr. Simard is Professor and Head of the Department of Biochemistry & Medical Genetics at the University of Manitoba in Winnipeg, Canada. Dr. Simard’s group demonstrated that SMN protein is abundant in neuronal growth cones, and most recently they have published work on the use of SMN mRNA as a biomarker in SMA clinical trials. She joined the SAB in 2005.

Stephen Strittmatter, M,D., PhD.
Dr. Strittmatter is the Vincent Coates Professor of Neurology and Neurobiology at Yale University School of Medicine. Dr. Strittmatter is a leading expert in the molecular mechanisms of axon regeneration in the adult CNS and periphery, including after spinal cord injury, publishing over 100 research articles in these fields.  Dr. Strittmatter joined the SAB in 2005.  Please click here to see his Yale University webpage.