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المؤلفMobini, Moein
المؤلفRadbakhsh, Shabnam
المؤلفKubaski, Francyne
المؤلفEshraghi, Peyman
المؤلفVakili, Saba
المؤلفVakili, Rahim
المؤلفAbbasifard, Mitra
المؤلفJamialahmadi, Tannaz
المؤلفRajabi, Omid
المؤلفEmami, Seyed Ahmad
المؤلفTayarani-Najaran, Zahra
المؤلفRizzo, Manfredi
المؤلفEid, Ali H
المؤلفBanach, Maciej
المؤلفSahebkar, Amirhossein
تاريخ الإتاحة2023-05-13T13:57:13Z
تاريخ النشر2023-04-06
اسم المنشورCurrent Medicinal Chemistry
المعرّفhttp://dx.doi.org/10.2174/0929867330666230406102555
الاقتباسMobini, M., Radbakhsh, S., Kubaski, F., Eshraghi, P., Vakili, S., Vakili, R., ... & Sahebkar, A. (2023). Effects of Trehalose Administration in Patients with Mucopolysaccharidosis Type III. Current Medicinal Chemistry.‏
معرّف المصادر الموحدhttp://hdl.handle.net/10576/42658
الملخصMucopolysaccharidosis 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. 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). 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. 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.
اللغةen
الناشرEurope PMC
الموضوعLysosomal storage disease (LSD)
MPS III
Mucopolysaccharidosis IIIA
Mucopolysaccharidosis IIIB
Sanfilippo syndrome
Trehalose
العنوانEffects of Trehalose Administration in Patients with Mucopolysaccharidosis Type III.
النوعArticle
dc.accessType Abstract Only


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