Abstract Objective Recent advances in the understanding of neurodevelopmental disorders such as Rett syndrome (RTT) have enabled the discovery of novel therapeutic approaches that require formal clinical evaluation of efficacy. Clinical trial success depends on outcome measures that assess clinical features that are most impactful for affected individuals. To determine the top concerns in RTT and RTT-related disorders we asked caregivers to list the top caregiver concerns to guide the development and selection of appropriate clinical trial outcome measures for these disorders. Methods Caregivers of participants enrolled in the US Natural History Study of RTT and RTT-related disorders (n = 925) were asked to identify the top 3 concerning pro...
Background: Rett syndrome is a neurodevelopmental disorder mainly affecting females. It is principal...
Abstract Background Rett syndrome is a severe neurodevelopmental disorder associated with mutations ...
BackgroundEarly development appears normal in Rett syndrome (OMIM #312750) and may be more apparent ...
As part of a wider study to investigate the behavioral phenotype of a national sample of girls and w...
As part of a wider study to investigate the behavioral phenotype of a national sample of girls and w...
Rett syndrome (RTT) is a neurodevelopmental disorder which is characterized by an apparently normal ...
Aim The aim of this study was to identify characteristics of epilepsy in Rett syndrome (RTT), and re...
© 2017 The Author(s). Background: Rett syndrome is a severe neurodevelopmental disorder associated w...
As part of a wider study to investigate the behavioral phenotype of a national sample of girls and w...
Purpose: Rett syndrome (RTT) is a neurodevelopmental disorder that almost exclusively affects female...
Aim To describe a new clinical tool, the Rett Syndrome Motor Evaluation Scale (RESMES) and to assess...
International audienceObjective: Rett syndrome is an X-linked dominant neurodevelopmental disorder c...
Objective: Rett syndrome is an X-linked dominant neurodevelopmental disorder caused by mutations in ...
Background: Early development appears normal in Rett syndrome (OMIM #312750) and may be more apparen...
ObjectiveRett syndrome is a severe neurodevelopmental disorder affecting approximately one in 10,000...
Background: Rett syndrome is a neurodevelopmental disorder mainly affecting females. It is principal...
Abstract Background Rett syndrome is a severe neurodevelopmental disorder associated with mutations ...
BackgroundEarly development appears normal in Rett syndrome (OMIM #312750) and may be more apparent ...
As part of a wider study to investigate the behavioral phenotype of a national sample of girls and w...
As part of a wider study to investigate the behavioral phenotype of a national sample of girls and w...
Rett syndrome (RTT) is a neurodevelopmental disorder which is characterized by an apparently normal ...
Aim The aim of this study was to identify characteristics of epilepsy in Rett syndrome (RTT), and re...
© 2017 The Author(s). Background: Rett syndrome is a severe neurodevelopmental disorder associated w...
As part of a wider study to investigate the behavioral phenotype of a national sample of girls and w...
Purpose: Rett syndrome (RTT) is a neurodevelopmental disorder that almost exclusively affects female...
Aim To describe a new clinical tool, the Rett Syndrome Motor Evaluation Scale (RESMES) and to assess...
International audienceObjective: Rett syndrome is an X-linked dominant neurodevelopmental disorder c...
Objective: Rett syndrome is an X-linked dominant neurodevelopmental disorder caused by mutations in ...
Background: Early development appears normal in Rett syndrome (OMIM #312750) and may be more apparen...
ObjectiveRett syndrome is a severe neurodevelopmental disorder affecting approximately one in 10,000...
Background: Rett syndrome is a neurodevelopmental disorder mainly affecting females. It is principal...
Abstract Background Rett syndrome is a severe neurodevelopmental disorder associated with mutations ...
BackgroundEarly development appears normal in Rett syndrome (OMIM #312750) and may be more apparent ...