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Top caregiver concerns in Rett syndrome and related disorders: data from the US natural history study
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...
Autores principales: | , , , , , , , |
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Formato: | Online Artículo Texto |
Lenguaje: | English |
Publicado: |
BioMed Central
2023
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Materias: | |
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 |