Neurobiologically-based treatments in Rett syndrome: opportunities and challenges

Hier ist eine Auswahl von Informationen von Patienten & Fachleuten, um die Rett-Syndrom besser verstehen zu können.

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During the last decade, there has been a dramatic increase in research aimed at treating neurodevelopmental disorders. These efforts have been the consequence of a better understanding of the genetic basis of several of these disorders and the subsequent development of experimental models, primarily mouse models. Although genetic treatment strategies have been successful in the laboratory setting, their clinical application is still hypothetical. Consequently, most of the emphasis on novel treatment development has focused on pharmacological approaches targeting processes downstream to the primary genetic abnormality.