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Efficacy of phosphodiesterase‐4 inhibitors in juvenile Batten disease (CLN3)

OBJECTIVE: Juvenile neuronal ceroid lipofuscinosis (JNCL), or juvenile Batten disease, is a pediatric lysosomal storage disease caused by autosomal recessive mutations in CLN3, typified by blindness, seizures, progressive cognitive and motor decline, and premature death. Currently, there is no treat...

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Detalles Bibliográficos
Autores principales: Aldrich, Amy, Bosch, Megan E., Fallet, Rachel, Odvody, Jessica, Burkovetskaya, Maria, Rama Rao, Kakulavarapu V., Cooper, Jonathan D., Drack, Arlene V., Kielian, Tammy
Formato: Online Artículo Texto
Lenguaje:English
Publicado: John Wiley and Sons Inc. 2016
Materias:
Acceso en línea:https://www.ncbi.nlm.nih.gov/pmc/articles/PMC5215570/
https://www.ncbi.nlm.nih.gov/pubmed/27804148
http://dx.doi.org/10.1002/ana.24815