A randomized controlled trial of Everolimus for neurocognitive symptoms in PTEN hamartoma tumor syndrome

PTEN hamartoma tumor syndrome (PHTS) is a complex neurodevelopmental disorder characterized by mTOR (mechanistic target of rapamycin) overactivity. Limited data suggest that mTOR inhibitors may be therapeutic. No placebo-controlled studies have examined mTOR inhibition on cognition and behavior in h...

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Veröffentlicht in:Human molecular genetics 2022-10, Vol.31 (20), p.3393-3404
Hauptverfasser: Srivastava, Siddharth, Jo, Booil, Zhang, Bo, Frazier, Thomas, Gallagher, Anne Snow, Peck, Fleming, Levin, April R, Mondal, Sangeeta, Li, Zetan, Filip-Dhima, Rajna, Geisel, Gregory, Dies, Kira A, Diplock, Amelia, Eng, Charis, Hanna, Rabi, Sahin, Mustafa, Hardan, Antonio
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Sprache:eng
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