| Budget Amount *help |
¥4,160,000 (Direct Cost: ¥3,200,000、Indirect Cost: ¥960,000)
Fiscal Year 2015: ¥1,040,000 (Direct Cost: ¥800,000、Indirect Cost: ¥240,000)
Fiscal Year 2014: ¥1,430,000 (Direct Cost: ¥1,100,000、Indirect Cost: ¥330,000)
Fiscal Year 2013: ¥1,690,000 (Direct Cost: ¥1,300,000、Indirect Cost: ¥390,000)
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| Outline of Final Research Achievements |
Some patients with myasthenia gravis (MG) caused by antibodies against muscle-specific kinase (MuSK; MuSK-MG) apparently do not respond to immunosuppressive therapy and rapidly progress to life-threatening muscle atrophy. In addition, long-term administration of corticosteroids, which are first-line treatment of MG, is frequently responsible for severe adverse effect. Therefore, new effective and safe immunosuppressive drugs are urgently required.
Here, we demonstrated that administration of rapamycin is able to act as a highly immunosuppressive drug in animal model of MuSK-MG and consequently improves clinical characteristics by maintaining NMJ structures and function. Moreover, our data strengthen the case for using rapamycin as a treatment of human MuSK-MG, although further large clinical trails are required to determine the exact use of rapamycin as a sole therapeutic or as an adjunctive treatment to other immunosuppressive drugs.
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