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 partic...
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Wiley,
2024-04-01T00:00:00Z.
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