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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...
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 |
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Formato: | Online Artículo Texto |
Lenguaje: | English |
Publicado: |
John Wiley and Sons Inc.
2016
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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 |
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