| Project/Area Number |
26870379
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| Research Category |
Grant-in-Aid for Young Scientists (B)
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| Allocation Type | Multi-year Fund |
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| Research Field |
Embryonic/Neonatal medicine
Pediatrics
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| Research Institution | Shimane University |
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Principal Investigator |
Yamada Kenji 島根大学, 医学部, 助教 (70624930)
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| Project Period (FY) |
2014-04-01 – 2016-03-31
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| Project Status |
Completed (Fiscal Year 2015)
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| Budget Amount *help |
¥4,030,000 (Direct Cost: ¥3,100,000、Indirect Cost: ¥930,000)
Fiscal Year 2015: ¥2,470,000 (Direct Cost: ¥1,900,000、Indirect Cost: ¥570,000)
Fiscal Year 2014: ¥1,560,000 (Direct Cost: ¥1,200,000、Indirect Cost: ¥360,000)
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| Keywords | ベザフィブラート / 脂肪酸代謝異常症 / グルタル酸血症2型 / in vitro probe assay |
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| Outline of Final Research Achievements |
Bezafibrate (BEZ) was reported as a drug for mild form of fatty acid oxidation disorders, but was considered not effective for severe (neonatal-onset) form. In this study, we investigated the efficacy of BEZ for fibroblasts derived from various form of glutaric acidemia type 2 (GA2) using in vitro probe (IVP) assay.
Low-dose BEZ was not effective for severest form of GA2 in which polycystic kidney, Potter's face, and severe cardiomyopathy was seen soon after birth. By contrast, low-dose BEZ was effective for neonatal-onset form without anomaly and cardiomyopathy. Moreover, good response to low-dose BEZ was observed in milder form which is onset after infancy. By the way, high-dose BEZ can not be evaluated using IVP assay due to precision of analysis
BEZ may be clinically effective even for neonatal-onset form of GA2 if the patient has no anomaly and cardiomyopathy.
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