Pitt Pediatrics congratulates Deepa Rajan, MD for their R61-R33 grant award from the National Institute of Health for their work “Database Harmonization for Creation and Validation of Outcome Assessments and Prediction Tools for Metachromatic Leukodystrophy (MLD)”.
The UPMC Children’s Center for NeuroGenomics(CCNG) lead by Rajan will partner with the Global Leukodystrophy Initiative (GLIA) Clinical Trial Network to create the largest harmonized MLD-database to date. Their goal is to harmonize and curate the database at Pitt, initiated by Maria Escolar, MD, MS at the Program for the Study of Neurodevelopment in Rare Disorders (NDRD) with the GLIA-MLD, along with the 9 major leukodystrophy centers to create the largest MLD database to date. They also want to make a rigorous Natural History platform will inform future clinical trials and be capable for use as non-concurrent control arm.
This grant will help create an accessible computing platform for supporting novel collaborations. In addition to an interactive academic research database, it will create publicly accessible computing platform for research transparency that will be made broadly accessible to researchers as part of the R61 phase of this proposal. In the R33 phase, Rajan’s team at the CCNG will work with the CHOP team to validate clinical outcome assessments (COAs) in new populations using the GLIA-MLD platform. This will also help create and validation of a clinical tool for MLD risk prediction.
Please join us in congratulating Deepa Rajan and her team for their grant and look forward to updates and stories about their research on our BlueSky and Instagram pages.