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You are here: Home > Research > Krabbe Translational Research Network (KTRN)

Krabbe Translational Research Network (KTRN)

KTRN Group Photo

The last few decades have seen an explosion of scientific and technological breakthroughs. Unfortunately, patient care has not kept up with the rapid pace of new discoveries. The goal of translational research is to break down barriers between disciplines to quickly translate basic research discoveries into practical applications. This "bench-to-beside" approach requires basic scientists and clinicians to work more closely, with information exchanged in both directions. Community input keeps the research focused on issues that matter most to patients and their families.

The Krabbe Translation Research Network (KTRN) is a consortium of scientists and clinicians who are dedicated to helping children with Krabbe disease live longer and healthier lives. The KTRN brings together the complementary knowledge and skills of investigators from different institutions and working in different disciplines to accelerate the development of research findings into new treatments.

KTRN members share data and resources to work towards a single goal. Together, we have identified promising therapeutic approaches for further development. To that end, members are working on collaborative projects and independent but interrelated studies.

The fifth annual meeting of the Krabbe Translation Research Network was held February 3‐5, 2016, Captiva Island, FL. This year's meeting focused on organizing preclinical studies that will be performed next year. The planned studies will move the initial proof of concept into larger animal models, refine biomarkers (e.g., psychosine and DTI-tractography studies), and continue studying the relationship between symptoms and disease mechanisms. Lastly, the first pathological studies of the brain in treated and untreated patients were completed thanks to the efforts of our newly formed subcommittee on tissue banking.

Summaries of annual meetings:

The Legacy of Angels Foundation has provided funding for KTRN meetings and the following projects:

Dr. Maria Escolar:

  • "Siemens MRI unit for newborn screening"
  • "Krabbe Translational Research Network collaborative lab grant"
  • "MRI for Program for the Study of Neurodevelopment in Rare Disorders"
  • "A virtual medical home and clinic for children with Krabbe disease"
  • "Program for the Study of Neurodevelopment in Rare Disorders"
  • "Roadmap Initiative to Support Gene Therapy for Krabbe’s Disease"

Sponsored by
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The Legacy of Angels