Defining pathways for development of disease-modifying therapies in children with type 1 diabetes: a consensus report.

Wherrett, Diane K; Chiang, Jane L; Delamater, Alan M; DiMeglio, Linda A; Gitelman, Stephen E; Gottlieb, Peter A; Herold, Kevan C; Lovell, Daniel J et al. · Diabetes Care · 2015

review · Level V

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Abstract

Emerging data suggest that type 1 diabetes is a more aggressive disease in children than in adults, with important differences in pathophysiology and clinical course. Therefore, the efficacy of disease-modifying therapies may be different in the two populations. Understanding the developmental and regulatory pathways for type 1 diabetes-modifying therapies in children will enable industry, academia, funders, advocacy groups, and regulators to translate new science to clinical care. This consensus report characterizes the fundamental differences in type 1 diabetes between children and adults and proposes a thoughtful approach to better understand the development and regulatory pathways for type 1 diabetes therapies.

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