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    Effects of Trehalose Administration in Patients with Mucopolysaccharidosis Type III

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    Date
    2023-06-21
    Author
    Mobini, Moein
    Radbakhsh, Shabnam
    Kubaski, Francyne
    Eshraghi, Peyman
    Vakili, Saba
    Vakili, Rahim
    Abbasifard, Mitra
    Jamialahmadi, Tannaz
    Rajabi, Omid
    Emami, Seyed Ahmad
    Tayarani-Najaran, Zahra
    Rizzo, Manfredi
    Eid, Ali H.
    Banach, Maciej
    Sahebkar, Amirhossein
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    Abstract
    Background and Aim: Mucopolysaccharidosis type III (MPS III) is a rare autosomal recessive lysosomal storage disease (LSD) caused by a deficiency of lysosomal enzymes required for the catabolism of glycosaminoglycans (GAGs), mainly in the central nervous system. Trehalose has been proposed as a potential therapeutic agent to attenuate neuropathology in MPS III. We conducted a single-arm, open-label study to evaluate the efficacy of trehalose treatment in patients with MPS IIIA and MPS IIIB. Methods: Five patients with MPS III were enrolled. Trehalose was administrated intravenously (15 g/week) for 12 weeks. Health-related quality of life and cognitive function, serum biomarkers, liver, spleen, and lung imaging were assessed to evaluate trehalose efficacy at baseline and trial end (week 12). Results: TNO-AZL Preschool children Quality of Life (TAPQOL) scores increased in all patients, and the mean scores for quality of life were increased after the intervention. Serum GAG levels were reduced in all treated patients (however, the differences were not statistically significant). Alanine aminotransferase (ALT) levels were reduced in all patients post-treatment (p=0.0039). The mean levels of aspartate transaminase (AST) were also decreased after 12 weeks of treatment with Trehalose. Decreased serum pro-oxidant-antioxidant balance and increased GPX activity were observed at the end of the study. Decreases in mean splenic length were observed, whereas the liver volume did not change. Conclusion: Improvements in health-related quality of life and serum biomarkers (GAGs, liver aminotransferase levels, antioxidant status), as well as liver and spleen size, were found following 3 months of trehalose administration in patients with MPS IIIA and MPS IIIB.
    URI
    https://www.scopus.com/inward/record.uri?partnerID=HzOxMe3b&scp=85169908011&origin=inward
    DOI/handle
    http://dx.doi.org/10.2174/0929867330666230406102555
    http://hdl.handle.net/10576/64523
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    • Medicine Research [‎1768‎ items ]

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