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Formulation factors beyond concentration significantly impact efficacy

Perkins DO, Jeffries CD, Do KQ

Critically, both subjects and investigators were blinded to the group assignment, ensuring impartiality throughout the study

Mutations in C9orf72 result in aberrant RNA repeats that trap proteins such as TDP-43, causing RNA metabolism to be disrupted and toxic dipeptide repeat proteins (DPRs) to be produced

This study clarifies a critical variable in gerotherapeutics: the route and duration of administration do not simply alter the magnitude of a drugs efficacy

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