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Improving clinical trial readiness to accelerate development of new therapeutics for Rett syndrome
Rett syndrome is associated with severe functional impairments and many comorbidities, each in urgent need of treatments. Mutations in the MECP2 gene were identified as causing Rett syndrome in 1999. Over the past 20 years there has been an abundance of preclinical research with some studies leading...
| Autores principales: | , , , , , |
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| Formato: | Online Artículo Texto |
| Lenguaje: | English |
| Publicado: |
BioMed Central
2022
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| Materias: | |
| Acceso en línea: | https://www.ncbi.nlm.nih.gov/pmc/articles/PMC8894842/ https://www.ncbi.nlm.nih.gov/pubmed/35246185 http://dx.doi.org/10.1186/s13023-022-02240-w |