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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Shranjeno v:
Bibliografske podrobnosti
izdano v:Ann Neurol
Main Authors: Aldrich, Amy, Bosch, Megan E., Fallet, Rachel, Odvody, Jessica, Burkovetskaya, Maria, Rama Rao, Kakulavarapu V., Cooper, Jonathan D., Drack, Arlene V., Kielian, Tammy
Format: Artigo
Jezik:Inglês
Izdano: John Wiley and Sons Inc. 2016
Teme:
Research Articles
Online dostop:https://ncbi.nlm.nih.gov/pmc/articles/PMC5215570/
https://ncbi.nlm.nih.gov/pubmed/27804148
https://ncbi.nlm.nih.govhttp://dx.doi.org/10.1002/ana.24815
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