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The CLN3 Disease Staging System: A new tool for clinical research in Batten disease.
Neurology. 2020 Jun 9;94(23):e2436-e2440. doi: 10.1212/WNL.0000000000009454. Epub 2020 Apr 16.
Neurology. 2020.
PMID: 32300063
Free PMC article.
Batten disease: an expert update on agents in preclinical and clinical trials.
Masten MC, Mink JW, Augustine EF.
Masten MC, et al.
Expert Opin Investig Drugs. 2020 Dec;29(12):1317-1322. doi: 10.1080/13543784.2020.1837110. Epub 2020 Nov 1.
Expert Opin Investig Drugs. 2020.
PMID: 33135495
Free PMC article.
No abstract available.
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A diagnostic confidence scheme for CLN3 disease.
Masten MC, Corre C, Paciorkowski AR, Vierhile A, Adams HR, Vermilion J, Zimmerman GA, Augustine EF, Mink JW.
Masten MC, et al.
J Inherit Metab Dis. 2021 Nov;44(6):1453-1462. doi: 10.1002/jimd.12429. Epub 2021 Sep 7.
J Inherit Metab Dis. 2021.
PMID: 34453334
Free PMC article.
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Challenges of Phenotype-Genotype Correlations in Rare Diseases.
Masten MC, Augustine EF, Mink JW.
Masten MC, et al.
JAMA Ophthalmol. 2021 Dec 1;139(12):1323. doi: 10.1001/jamaophthalmol.2021.4372.
JAMA Ophthalmol. 2021.
PMID: 34709366
No abstract available.
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