Advancing clinical development for neuronopathic Hunter syndrome through a quantitatively-driven reverse translation framework.

ABSTRACT: A quantitatively-driven evaluation of existing clinical data and associated knowledge to accelerate drug discovery and development is a highly valuable approach across therapeutic areas, but remains underutilized. This is especially the case for rare diseases for which development is particularly challenging. The current work outlines an organizational framework to support a quantitatively-based reverse translation approach to clinical development. This approach was applied to characterize predictors of the trajectory of cognition in Hunter syndrome (Mucopolysaccharidosis Type II; MPS-II), a rare X-linked lysosomal storage disorder, highly heterogeneous in its course. Specifically, we considered ways to refine target populations based on age, cognitive status, and biomarkers, that is, cere

SUBMITTER: Latzman RD 

PROVIDER: S-EPMC10974710 | biostudies-literature | 2024 Apr

REPOSITORIES: biostudies-literature