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Dynamic Research Infrastructure: Adeline Vanderver, MD

Published on · Last Updated 1 year 6 months ago


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The Leukodystrophy Program is an example of a lab that has grown into a dynamic research infrastructure.


The Leukodystrophy Center at CHOP is fairly young and was really driven, before I got here, by a parent who faced a diagnosis here at CHOP for which there was no therapy and for which there was really no hope of anything but endless doctors’ appointments and potential loss of life. And that parent worked with teams here at CHOP. To explain to people how pivotal this group of disease is, it’s estimated that one in 6,000 children are born with a leukodystrophy. How important this disease community was to CHOP and to its central mission of enhancing the health and well-being of children everywhere including children with rare disease.

That parent and thoughtful people here at CHOP in leadership really energized the creation of the Leukodystrophy Program. And in the five years since that time, the Leukodystrophy Program has grown to include dozens of clinical providers that provide clinical care to hundreds of children with leukodystrophies from all around the country and the world, every year. But has also grown to a very dynamic research infrastructure which includes everything from the patient to the therapy and back to the patient, involving multi-models for testing therapeutics and for testing diagnoses. And so I think that if I think about my team and the overall group here, I would say it’s transformative.