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Editorial
. 2020 Dec;29(12):1317-1322.
doi: 10.1080/13543784.2020.1837110. Epub 2020 Nov 1.

Batten disease: an expert update on agents in preclinical and clinical trials

Margaux C Masten  1 , Jonathan W Mink  1 , Erika F Augustine  1
Affiliations
Editorial

Batten disease: an expert update on agents in preclinical and clinical trials

Margaux C Masten et al. Expert Opin Investig Drugs. 2020 Dec.
No abstract available

Keywords: Batten disease; Disease-modifying therapy; gene therapy; neurodegenerative diseases; neuronal ceroid lipofuscinosis; pediatric.

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References

    1. Augustine E, Mink J. Neuronal Ceroid Lipofuscinosis. 2020. April 17, 2020 [cited April 24, 2020]; Available from:

    1. Johnson TB, Cain JT, White KA, Ramirez-Montealegre D, Pearce DA, Weimer JM. Therapeutic landscape for Batten disease: current treatments and future prospects. Nat Rev Neurol 2019. March;15(3):161–78.

      This is an excellent comprehensive review of the variety of approaches currently in pre-clinical and clinical testing for treatment of the NCLs.

    1. Palmieri M, Pal R, Nelvagal HR, Lotfi P, Stinnett GR, Seymour ML, et al. mTORC1-independent TFEB activation via Akt inhibition promotes cellular clearance in neurodegenerative storage diseases. Nature communications 2017. February 6;8:14338. - PMC - PubMed
    1. Cendret V, Legigan T, Mingot A, Thibaudeau S, Adachi I, Forcella M, et al. Synthetic deoxynojirimycin derivatives bearing a thiolated, fluorinated or unsaturated N-alkyl chain: identification of potent α-glucosidase and trehalase inhibitors as well as F508del-CFTR correctors. Organic & Biomolecular Chemistry 2015;13(43):10734–44. - PubMed
    1. Ghosh A, Rangasamy SB, Modi KK, Pahan K. Gemfibrozil, food and drug administration-approved lipid-lowering drug, increases longevity in mouse model of late infantile neuronal ceroid lipofuscinosis. J Neurochem 2017. May;141(3):423–35. - PMC - PubMed

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