News Release

Wednesday, September 9, 2009

NINDS Names Dr. Petra Kaufmann Director of the Office of Clinical Research

The National Institute of Neurological Disorders and Stroke (NINDS), part of the National Institutes of Health, has named Petra Kaufmann, M.D., M.Sc., as director of its Office of Clinical Research.

Dr. Kaufmann is among the foremost experts in the design and management of clinical trials for neuromuscular disorders, including spinal muscular atrophy (SMA), amyotrophic lateral sclerosis (ALS), and mitochondrial diseases. In her new role, Dr. Kaufmann will lead the Institute’s efforts to increase the effectiveness of clinical studies by addressing issues such as optimal trial design, ethical safe conduct of trials, and challenges in patient enrollment.

"Dr. Kaufmann has experience in all phases of clinical research, from conducting laboratory investigation and studies on disease mechanism to serving in key leadership positions on several major multicenter trials," said Story C. Landis, Ph.D., director of NINDS. "Dr. Kaufman's outstanding skills and expertise will allow us to make the most of the scientific opportunities ahead and to have a significant impact on clinical neuroscience."

Dr. Kaufmann said, "I look forward to supporting excellence in clinical research at NINDS so that the advances in neuroscience can be translated into better treatments for patients."

Prior to her appointment, Dr. Kaufmann was co-director of the SMA Clinical Research Center and an associate professor of neurology at Columbia University's Neurological Institute, where she has been a faculty member since 2000. She was also director of Columbia's clinical trials unit within the Pediatric Neuromuscular Clinical Research Network for SMA. She served as co-principal investigator for two NINDS-funded multicenter clinical trials to treat ALS, also known as Lou Gehrig's disease. In addition, she was co-investigator for studies on periodic paralyses and Duchenne muscular dystrophy.

Throughout her career, Dr. Kaufmann has implemented novel tools and techniques in clinical trials, including the development of Web-based data management systems, telephone-administered neurological scales, imaging tests in ALS, and magnetic resonance spectroscopy to measure a biomarker in mitochondrial disorders.

Her many contributions to clinical research have had considerable impact. Findings from her work on mortality and disease progression in SMA influenced NINDS policy in reshaping aspects of its clinical trials for a number of diseases. She was principal investigator on an NINDS-funded trial to treat ALS with Coenzyme Q10, an antioxidant. Although the study results did not support a beneficial effect of the therapy, Dr. Kaufmann's innovative design strategy led to the trial’s efficient conclusion in 2009.

Dr. Kaufmann's 2009 study on mitochondrial encephalopathy with lactic acidosis and stroke (MELAS) helped advance understanding of the effects of a genetic mutation associated with this disease. The research points the way toward better screening and diagnosis of patients with the mutation, which also has been found in some people with autism spectrum disorder.

In 2007, Dr. Kaufmann was elected to the American Neurological Association, where she serves on the Scientific Program Committee. In addition, she is a member of numerous other professional societies, including the American Academy of Neurology, the American Society for Experimental NeuroTherapeutics, and the World Federation of Neurology. Dr. Kaufmann has published more than 45 peer-reviewed articles and has written or edited dozens of review articles and book chapters.

Dr. Kaufmann was born in Hersel, Germany. She earned her medical degree from the University of Bonn, Germany. She developed an early interest in neuromuscular diseases and conducted part of her medical training at the field's premiere research programs in London and Paris. In 1993 she joined Columbia University as a postdoctoral fellow studying the molecular genetics of mitochondrial disease. She completed an internship in medicine at St. Luke's-Roosevelt Hospital Center in New York and a neurology residency at Columbia. She also earned her masters in science degree in biostatistics from Columbia’s Mailman School of Public Health.

The NINDS ( is the nation's leading funder of research on the brain and nervous system. The NINDS mission is to reduce the burden of neurological disease — a burden borne by every age group, by every segment of society, by people all over the world. For more information about the NINDS Office of Clinical Research, visit

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