A framework for translating tauopathy therapeutics: Drug discovery to clinical trials.

ABSTRACT: The tauopathies are defined by pathological tau protein aggregates within a spectrum of clinically heterogeneous neurodegenerative diseases. The primary tauopathies meet the definition of rare diseases in the United States. There is no approved treatment for primary tauopathies. In this context, designing the most efficient development programs to translate promising targets and treatments from preclinical studies to early-phase clinical trials is vital. In September 2022, the Rainwater Charitable Foundation convened an international expert workshop focused on the translation of tauopathy therapeutics through early-phase trials. Our report on the workshop recommends a framework for principled drug development and a companion lexicon to facilitate communication focusing on reproducibility a

SUBMITTER: Feldman HH 

PROVIDER: S-EPMC11567863 | biostudies-literature | 2024 Nov

REPOSITORIES: biostudies-literature