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Search Magazine
August 08, 2018
Patient by patient: Finding cures for rare diseases
Four years ago as part of The Burgess Lab at The Jackson Laboratory, Kathy Morelli and a large team of collaborators took on the challenge of creating a cure for Caroline, a young girl who was battling an incurable disease called Charcot-Marie-Tooth.
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A Cure for Caroline
JAX Professor Robert Burgess and collaborators are developing personalized gene therapy for a Texas child suffering from a neuromuscular disease.
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Download The Spring 2019 CMTA Report
The 2019 Spring Report from the Charcot-Marie-Tooth Association (CMTA) highlights work being done by JAX Professor Robert Burgess and collaborators toward a clinical trial for a young girl who has CMT type 2D, a rare and severe form of the disease.
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A rare disease calls for a special mouse model
For the rarest of rare genetic conditions, a one-of-a-kind mouse could light a path to new treatments.
