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Krabbe xTranslational Research Network (KTRN)

KTRN Group Photo

The fifth annual meeting of the Krabbe Translation Research Network was held March 3–6, 2015, Captiva Island, FL. This year’s meeting focused on patient advocacy, which is of critical importance in rare disease research. Micki Gartze was received a patient advocacy award for her many years of contributions to advocating for better detection and treatment of Krabbe disease.

It is understood that collaboration between researchers and families accelerates progress toward new treatments and improves the lives of affected children and their families. Families need a unified voice to effectively advocate for policies that benefit their children. To help parents gain a broad understanding of critical issues in treatment and research, researchers and clinicians who specialize in Krabbe disease, and professional in related fields gave presentations on the following topics:

Challenges faced by researchers Issues surrounding clinical trial data Detection and diagnosis of Krabbe disease Progress in the development of new therapies Collaboration with other sites Biorepositories The NDRD Virtual Medical Home

Subcommittees were established with to address the following issues: Biorepository: to determine the capacity to store tissues and data for patient-centered outcomes research and develop policies for sharing biological samples. Data structure and clinical trials: to identify clinical endpoints that are important to families, educate families about research, identify ways to improve network capacity to acquire comprehensive and complete longitudinal data on patients, and develop a platform for participating researchers to obtain reports/analyses. Early detection and diagnosis: to develop a consensus statement about this issue to be shared with policy makers and other stakeholders. Patient engagement: to expand the network by recruiting a broader, more representative population and improve real-time data collection using standardized validated outcomes instruments when possible.

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