To develop a multi-disciplinary approach to understand how myelin is formed, how axons and myelin are damaged in disease, and how they may be repaired.
- To study myelin formation as a source for strategies of myelin repair
- To study consequences of myelin damage—common to all diseases of myelin—such as inflammation or neurodegeneration
- To provide better cellular and animal models for identifying mechanisms of disease
- To exploit models to perform preclinical studies of therapies
- To translate the research of HJKRI into prognostic, diagnostic and treatment application
To provide better diagnostic, prognostic and therapeutic options for Krabbe Leukodystrophy patients.
- To develop initiatives to maximize the success of the Newborn Screening program for Krabbe Disease
- To increase understanding of various types of Krabbe Disease, determine which biochemical, genetic, and neurodiagnostic tests predict phenotype
- To educate physicians about Krabbe Disease and Newborn Screening
- To develop natural history studies, clinical evaluation and treatment protocols for Krabbe Disease