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Krabbe Translational Research Network (KTRN)

The first meeting of the Krabbe Translational Research Network (KTRN) was held October 10–12, 2010 i n Chapel Hill, North Carolina. The initial goal was to bring together clinicians and researchers specializing in Krabbe disease to work together to accelerate the development of improved treatments for Krabbe disease. At this meeting scientists who had never seen a patient with Krabbe disease exchanged information with the families of patients and clinicians from various disciplines. These collaborative interactions enable crucial observations about disease processes, l eading to exciting new discoveries, useful diagnostic tools, and novel therapies.

As the meeting progressed it became clear that there was a need to improve outcomes of the only treatment currently available, umbilical cord blood transplantation. For this purpose, a group of gene therapy experts and clinicians began a collaboration to develop a gene therapy approach that could address the limitations of umbilical cord blood transplantation. A pilot study involving Dr. Steven Gray, Dr. Tal Kafri, and Dr. David Wenger was set up to explore the best gene therapy vector for this purpose.

It was also decided that to be able to translate research findings into clinical practice the KTRN should pursue improved validated clinical endpoints and biomarkers, natural history data, additional preclinical therapy development, and the pre-planning of clinical trials.

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