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Future perspectives: Moving towards NCL treatments.


ABSTRACT: Clinicians, basic researchers, representatives from pharma and families from around the world met in Cordoba, Argentina in October, 2014 to discuss recent research progress at the 14th International Congress on Neuronal Ceroid Lipofuscinoses (NCLs; Batten disease), a group of clinically overlapping fatal, inherited lysosomal disorders with primarily neurodegenerative symptoms. This brief review article will provide perspectives on the anticipated future directions of NCL basic and clinical research as we move towards improved diagnosis, care and treatment of NCL patients. This article is part of a Special Issue entitled: Current Research on the Neuronal Ceroid Lipofuscinoses (Batten Disease).

SUBMITTER: Cotman SL 

PROVIDER: S-EPMC4567439 | biostudies-literature | 2015 Oct

REPOSITORIES: biostudies-literature

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Future perspectives: Moving towards NCL treatments.

Cotman Susan L SL   Mole Sara E SE   Kohan Romina R  

Biochimica et biophysica acta 20150407 10 Pt B


Clinicians, basic researchers, representatives from pharma and families from around the world met in Cordoba, Argentina in October, 2014 to discuss recent research progress at the 14th International Congress on Neuronal Ceroid Lipofuscinoses (NCLs; Batten disease), a group of clinically overlapping fatal, inherited lysosomal disorders with primarily neurodegenerative symptoms. This brief review article will provide perspectives on the anticipated future directions of NCL basic and clinical resea  ...[more]

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