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.
The Center for Pediatric Neurological Disease Research (CPNDR) is tasked with advancing basic research investigating complex, pressing questions relating to pediatric neurological diseases.
The Center for Experimental Neurotherapeutics (CENT) is designed to facilitate the rapid clinical development of promising treatments for catastrophic pediatric neurological diseases.
The Office of Strategy & Alliances (OSA) drives engagement and collaboration with external stakeholders to accelerate and maximize the impact of research activities conducted across PTNI
Participation in clinical trials with investigational drugs for pediatric neurologic and metabolic diseases
Development of outcome measures, biomarkers, and clinical trial design for pediatric neurologic diseases
Understanding the trajectories of change in pediatric neuromuscular diseases: spinal muscular atrophy, Duchenne muscular dystrophy, and Charcot-Marie-Tooth inherited neuropathies.
