Trofinetide Treatment Demonstrates a Benefit Over Placebo for the Ability to Communicate in Rett Syndrome

Rett syndrome: Revised diagnostic criteria and nomenclature

Jeffrey L. Neul, Walter E. Kaufmann, Daniel G. Glaze et al. · Annals of Neurology · 2010 · 1.4K citations · Full text

Life Sciences, Genetics, Neurology +17

Partial reversal of Rett Syndrome-like symptoms in MeCP2 mutant mice

Daniela Tropea, Emanuela Giacometti, Nathan R. Wilson et al. · Proceedings of the National Academy of Sciences · 2009 · 595 citations · Full text

Life Sciences, Neurobiology, Neuroscience +17

Validity and Reliability of the Communication and Symbolic Behavior Scales Developmental Profile With Very Young Children

Amy M. Wetherby, Lori Allen, Julie Cleary et al. · Journal of Speech Language and Hearing Research · 2002 · 247 citations

Social Sciences, Human Development, Psychology +15

The Rett Syndrome Behaviour Questionnaire (RSBQ): refining the behavioural phenotype of Rett syndrome

Rebecca H. Mount, Tony Charman, Richard P. Hastings et al. · Journal of Child Psychology and Psychiatry · 2002 · 189 citations

Medicine, Neurology, Psychology +6

Trofinetide for the treatment of Rett syndrome: a randomized phase 3 study

Jeffrey L. Neul, Alan K. Percy, Tim A. Benke et al. · Nature Medicine · 2023 · 186 citations · Full text

Medicine, Pharmacology, Pharmacotherapy +11