Top caregiver concerns in Rett syndrome and related disorders: data from the US natural history study

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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...

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Detalles Bibliográficos
Autores principales: Neul, Jeffrey L., Benke, Timothy A., Marsh, Eric D., Suter, Bernhard, Silveira, Lori, Fu, Cary, Peters, Sarika U., Percy, Alan K.
Formato: Online Artículo Texto
Lenguaje:English
Publicado: BioMed Central 2023
Materias:
Research
Acceso en línea:https://www.ncbi.nlm.nih.gov/pmc/articles/PMC10571464/
https://www.ncbi.nlm.nih.gov/pubmed/37833681
http://dx.doi.org/10.1186/s11689-023-09502-z
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