Global Leukodystrophy Initiative Clinical Trials Network (GLIA- CTN)

Children'S Hosp Of Philadelphia, Philadelphia PA

Investigators

Linked publications, trials & patents

Abstract

The Career Enhancement core is dedicated to the recruitment, support, and retention of qualified investigators and clinicians who will continue innovation in rare diseases such as the leukodystrophies. We propose formal mentorship and training opportunities in rare disease research to facilitate long-term commitment and retention. Aim 1 is to prepare pre-graduate students for a career in leukodystrophy and rare disease research. We will leverage programs supporting pre-doctoral mentorship and education: Translational Research Immersion Program (TRIP) and the leukodystrophy specific Predoctoral Preparatory Program (P3). The expected outcome of this Aim is an increased pipeline for future rare disease researchers needed to address clinical care needs in care disease. Aim 2 will be to support translational research scientist development in leukodystrophy research. Two leukodystrophy fellowships are offered, the Mass General-Brigham (MGB) Neurogenetics and Gene Therapy Fellowship and The Children’s Hospital of Philadelphia (CHOP) Leukodystrophy Fellowship, in addition to offering career development awards to early-stage investigators and those who are new to leukodystrophy research. The expected outcome is an increased retention of early-stage investigators in rare disease clinical research. Aim 3 is to support faculty development with focus on communities with high unmet need. We will develop a leukodystrophy-specific communication training program using the VitalTalk® platform and support the Leukodystrophy Education and Access Program (GLIA-LEAP). The expected outcome is an increased engagement with communities without access to leukodystrophy clinical care and translational research, including those at a geographic distance from tertiary care centers. Overall, the GLIA-CDC hopes to create a leukodystrophy training pipeline, engaging clinical scientists from undergraduate to postgraduate, in order to meet the emerging needs in diagnosis and therapy of this community.