About PTNI

Catastrophic neurological disorders in children are among the greatest medical challenges of our time. Many of these have a genetic contribution, often driven by a single gene mutation, particularly in pediatric cases. While tremendous advances in our understanding of the molecular basis of many of these diseases have been made, and symptom management and palliative therapies have improved survival, the underlying causes and clinical trajectory for many of these disorders remain to be addressed. A huge unmet need exists today for the majority of these disorders where no effective treatments are currently available, especially in those that are progressive and disabling in childhood. There is a unique opportunity to accelerate the development of novel therapies for seriously ill children living with these neurological disorders. The Pediatric Translational Neuroscience Initiative (PTNI) leverages the scientific strength and expertise in experimental therapeutics of St. Jude to rise to the challenge of altering the landscape of pediatric neurological disease.


PTNI programs


PTNI Faculty