Neuron-derived extracellular vesicles to examine brain mTOR target engagement with sirolimus in patients with multiple system atrophy.
Pucha KA; Ma TC; York W; Kang UJ; Kaufmann H; Kapogiannis D; Palma JA · 2023 · Parkinsonism & related disorders
WASTE classifies this as Negative / Null Result Report · AI classification, approximate
The study found no significant effect — useful as a negative control or null benchmark for your own design.
Abstract (excerpt)
Introduction Impaired autophagy is a pathogenic mechanism in the synucleinopathies. Sirolimus, a potent mTOR inhibitor and autophagy activator, had no beneficial effects in a randomized placebo-controlled trial in patients with multiple…
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Metadata source: Europe PMC · DOI 10.1016/j.parkreldis.2023.105821