title: Progress in the Development of Small Molecule Therapeutics for the Treatment of Neuronal Ceroid Lipofuscinoses (NCLs).
abstract: The neuronal ceroid lipofuscinoses (NCLs) are a group of inherited and incurable neurodegenerative disorders primarily afflicting the pediatric population. Current treatment regimens offer only symptomatic relief and do not target the underlying cause of the disease. Although the underlying pathophysiology that drives disease progression is unknown, several small molecules have been identified with diverse mechanisms of action that provide promise for the treatment of this devastating disease. This review aims to summarize the current cellular and animal models available for the identification of potential therapeutics and presents the current state of knowledge on small molecule compounds that demonstrate in vitro and/or in vivo efficacy across the NCLs with an emphasis on targets of action.
authors.affiliation: Department of Pharmaceutical Sciences, School of Pharmacy, Texas Tech University Health Sciences Center , Amarillo, Texas 79106, United States.
authors.forename: Nihar
authors.initials: N
authors.lastname: Kinarivala
authors.affiliation: Department of Pharmaceutical Sciences, School of Pharmacy, Texas Tech University Health Sciences Center , Amarillo, Texas 79106, United States.|Center for Chemical Biology, Department of Chemistry and Biochemistry, Texas Tech University , Lubbock, Texas 79409, United States.
authors.forename: Paul C
authors.initials: PC
authors.lastname: Trippier
mesh_terms: D000818:Animals; D006801:Humans; D015394:Molecular Structure; D009472:Neuronal Ceroid-Lipofuscinoses / Q000188:drug therapy*; D054852:Small Molecule Libraries / Q000737:chemistry / Q000627:therapeutic use*
chemical_list: D054852:Small Molecule Libraries