Double-blind, randomized, placebo-controlled study of trofinetide in pediatric Rett syndrome

To determine safety, tolerability, and pharmacokinetics of trofinetide and evaluate its efficacy in female children/adolescents with Rett syndrome (RTT), a debilitating neurodevelopmental condition for which no pharmacotherapies directed at core features are available. This was a phase 2, multicente...

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Bibliographic Details
Published in:	Neurology Vol. 92; no. 16; p. e1912
Main Authors:	Glaze, Daniel G, Neul, Jeffrey L, Kaufmann, Walter E, Berry-Kravis, Elizabeth, Condon, Sean, Stoms, George, Oosterholt, Sean, Della Pasqua, Oscar, Glass, Larry, Jones, Nancy E, Percy, Alan K
Format:	Journal Article
Language:	English
Published:	United States 16.04.2019
Subjects:	Adolescent Anti-Inflammatory Agents, Non-Steroidal - adverse effects Anti-Inflammatory Agents, Non-Steroidal - pharmacokinetics Anti-Inflammatory Agents, Non-Steroidal - therapeutic use Child Child, Preschool Double-Blind Method Female Glutamates - adverse effects Glutamates - pharmacokinetics Glutamates - therapeutic use Glutamic Acid - adverse effects Glutamic Acid - pharmacokinetics Glutamic Acid - therapeutic use Humans Rett Syndrome - drug therapy Treatment Outcome
ISSN:	1526-632X, 1526-632X
Online Access:	Get more information
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