Comparison of Core Features in Four Developmental Encephalopathies in the Rett Natural History Study.

Clare Cutri-FrenchDallas ArmstrongJoni SabyCasey GormanJane LaneCary FuSarika U PetersAlan PercyJeffrey Lorenz Neul Eric D Marsh

Published in: Annals of neurology (2020)

Although these developmental encephalopathies share many clinical features, clear differences in severity, regression, and seizures warrant considering them as unique disorders. These results will aid in the development of disease-specific severity scales, precise therapeutics, and future clinical trials. ANN NEUROL 2020;88:396-406.

Keyphrases

clinical trial
small molecule
current status
randomized controlled trial
phase ii
open label
study protocol