Leading Medical Institutions Establish a Clinical Research Network to Study SMA

New York, NY – September 24, 2004 – Researchers at Columbia University, Children’s Hospital Boston and The Children’s Hospital of Philadelphia have established a clinical research network to study spinal muscular atrophy (SMA), the leading genetic killer of infants and toddlers. The Pediatric Neuromuscular Clinical Research Network will address the vital need in SMA research to rapidly assess potential new therapeutic treatments in Phase I and Phase II clinical studies.

Headquartered at Columbia University Medical Center, the research network will be led by program director, Darryl De Vivo, M.D. In addition to Dr. De Vivo, the network of researchers will include: Petra Kaufmann, M.D., M.Sc. (Assistant Professor of Neurology, Columbia University Medical Center), Basil Darras, M.D. (Professor of Neurology, Children’s Hospital Boston/Harvard Medical School) and Richard Finkel, M.D. (Associate Clinical Professor of Pediatrics and Neurology, The Children’s Hospital of Philadelphia/University of Pennsylvania School of Medicine). The research network will be further supported by researchers at the University of Rochester in Rochester, N.Y.

“Fast and flexible Phase I and II trials are a critical step in developing therapeutic options for orphan diseases such as SMA,” commented Dr. De Vivo, Sidney Carter Professor of Neurology at Columbia University Medical Center and Director of the SMA Clinic. “With no current treatments available for this devastating disease, this initiative is a major step in moving research forward to quickly assess new therapies and provide rapid access of data to clinical investigators across the country.”

The Pediatric Neuromuscular Clinical Research Network will launch with a natural history study of patients suffering from spinal muscular atrophy. This research initiative is currently in its planning stage – families interested in participating in this or any other studies conducted by the research network should ask their physician to contact the investigators listed above.

Loren Eng, Co-Founder and President of the SMA Foundation commented, “This network is a critical and necessary step in developing treatments for spinal muscular atrophy. Patients will have access to a high caliber cadre of experienced physicians led by Darryl De Vivo, one of the top pediatric neurologists in the world. Developers of SMA drug treatments will gain a critical drug development tool and resource – a high quality database of patients and a results-driven research network focused on rapidly bringing treatments to SMA patients.”

Initial funding for the Pediatric Neuromuscular Clinical Research Network is provided by the Spinal Muscular Atrophy Foundation, a nonprofit organization located in New York. The research network augments the Foundation’s aggressive agenda to fund research initiatives which will accelerate the development of a treatment or cure for SMA.

About Spinal Muscular Atrophy

Spinal muscular atrophy is a genetic, motor neuron disease characterized by the wasting of skeletal muscles. Caused by progressive degeneration of nerve cells in the spinal cord, the disease leads to increasing muscular weakness and atrophy. Spinal muscular atrophy is often likened to polio because the same spinal cord cells are attacked in each disease. Over time, its victims continue to lose muscle control and strength, leading to progressive inability to walk, stand, sit up and breathe.

About SMA Foundation

The SMA Foundation is a nonprofit organization founded in 2003 dedicated to finding a treatment and 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, education and increased federal funding and support. For more information on the Spinal Muscular Atrophy Foundation, visit www.smafoundation.org or call (646) 253-7100.

# # #

Media Contact:
Bryan deCastro
(631) 495-9177
[email protected]

Cynthia Joyce
(646) 253-7100
[email protected]