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Amy T. Waldman

Dr. Waldman is an assistant professor of neurology at the Perelman School of Medicine at the University of Pennsylvania. Dr. Waldman received her medical doctorate from Jefferson Medical College (Thomas Jefferson University). She completed her pediatrics residency at The Children’s Hospital of Philadelphia (CHOP) and child neurology residency at both CHOP and the Hospital of the University of Pennsylvania. A recipient of the National Multiple Sclerosis Society-American Academy of Neurology Foundation (now the American Brain Foundation) Clinician Scientist Development Award, Dr. Waldman completed a fellowship in pediatric and adult MS at CHOP and the Hospital of the University of Pennsylvania. During her fellowship, she obtained a master of science in clinical epidemiology degree at Penn.

In 2005, she co-founded the Pediatric MS Center at CHOP, and in 2014, she co-founded the Leukodystrophy Center of Excellence at The Children’s Hospital of Philadelphia where she now serves as the medical director. Dr. Waldman’s primary research focuses on the development and interpretation of outcome measures for clinical trials in neuroinflammatory and neurodegenerative diseases. She has received funding from the NIH to study visual function and axonal loss through imaging in children with multiple sclerosis. Her work has also highlighted differences between pediatric and adult-onset MS. She is also a site principal investigator for collaborative research projects with other pediatric MS centers throughout the United States and Canada. Her research has been published in Neurology, Multiple Sclerosis and Related Disorders, Multiple Sclerosis International, Journal of the American Association for Pediatric Ophthalmology and Strabismus, Journal of Child Neurology, and others. She has authored numerous book chapters on the clinical features, treatment, and prognosis of CNS demyelination.

Dr. Waldman’s K23 award, entitled Development of Visual and Neurologic Outcome Measures in Pediatric MS, provided the infrastructure and knowledge on clinical trial methodology for her current work in leukodystrophies. There is a lack of standardized metrics for use in neurodegenerative diseases and a critical need for expertise in developing such tools. A first-in-human clinical trial using antisense oligonucleotide in Alexander disease is imminent; however, there are no outcome measures that have been established for this disorder. Her position as medical director of the Leukodystrophy Center at CHOP has provided her with the opportunity to lead an international natural history study for Alexander disease.

Dr. Waldman will direct Project 3 and serve as a co-investigator for Project 1.