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Long-Term Evaluation of Biomarkers in the Czech Cohort of Gaucher Patients.
Int J Mol Sci. 2023 Sep 22;24(19):14440. doi: 10.3390/ijms241914440.
Int J Mol Sci. 2023.
PMID: 37833892
Free PMC article.
Trial of N-Acetyl-l-Leucine in Niemann-Pick Disease Type C.
Bremova-Ertl T, Ramaswami U, Brands M, Foltan T, Gautschi M, Gissen P, Gowing F, Hahn A, Jones S, Kay R, Kolnikova M, Arash-Kaps L, Marquardt T, Mengel E, Park JH, Reichmannová S, Schneider SA, Sivananthan S, Walterfang M, Wibawa P, Strupp M, Martakis K.
Bremova-Ertl T, et al. Among authors: reichmannova s.
N Engl J Med. 2024 Feb 1;390(5):421-431. doi: 10.1056/NEJMoa2310151.
N Engl J Med. 2024.
PMID: 38294974
Clinical Trial.
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