Article

Systematic Review of Patient Focused Drug Development Meeting Reports for Conditions Affecting Neurodevelopment

2025-11-13

Abstract excerpt

<p>Background: Researchers of rare diseases affecting neurodevelopment struggle with endpoint identification and selection issues that are uniquely associated with developmental concepts but common across conditions. However, the potential to capitalize on the collective commonness of rare disease to achieve the large samples often required by psychometric methodologies is insufficiently tapped. Our goal was to id...

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Literature Corpus work: 846e8c34-d252-551a-930a-7bde31e2191b
DOI: 10.31234/osf.io/kx3dt_v1

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