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...

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Detalles Bibliográficos
Autores principales: Leonard, Helen, Gold, Wendy, Samaco, Rodney, Sahin, Mustafa, Benke, Timothy, Downs, Jenny
Formato: Online Artículo Texto
Lenguaje:English
Publicado: BioMed Central 2022
Materias:
Review
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

Internet

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

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