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Article

Pharmacokinetics and clinical efficacy of 6'-sialyllactose in patients with GNE myopathy: Randomized pilot trial.

Biomedicine & pharmacotherapy = Biomedecine & pharmacotherapie - 1 Dec 2023

Park Young-Eun, Park Eunjung, Choi Jaeil, Go Hiroe, Park Dan Bi, Kim Min-Young, Sung Nam Ji, Kim Lila, Shin Jin-Hong

Abstract excerpt

GNE myopathy, caused by biallelic mutations in the GNE gene, is characterized by initial ankle dorsiflexor weakness and rimmed vacuoles in the muscle histopathology, resulting in reduced sialic acid production. Sialyllactose is a source of sialic acid. We performed a pilot clinical trial to analyze the pharmacokinetic properties of 6'-sialyllactose (6SL) and evaluated the safety, and efficacy of oral 6SL in...

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PMID: 37852099
DOI: 10.1016/j.biopha.2023.115689

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