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Study on the safety and effect of novel therapeutic agents for mucopolysaccharidosis

Research Project

Project/Area Number 26461523
Research Category

Grant-in-Aid for Scientific Research (C)

Allocation TypeMulti-year Fund
Section一般
Research Field Pediatrics
Research InstitutionGifu University

Principal Investigator

Orii Kenji  岐阜大学, 医学部附属病院, 准教授 (30332688)

Co-Investigator(Kenkyū-buntansha) 折居 忠夫  岐阜大学, 大学院医学系研究科, 非常勤講師 (20045339)
戸松 俊治  岐阜大学, 大学院医学系研究科, 非常勤講師 (70237105)
鈴木 康之  岐阜大学, 医学部, 教授 (90154559)
Co-Investigator(Renkei-kenkyūsha) TOMATSU SHUNJI  岐阜大学, 大学院医学系研究科, 非常勤講師 (70237105)
Research Collaborator SUZUKI YASUYUKI  岐阜大学, 医学部, 教授 (90154559)
Project Period (FY) 2014-04-01 – 2017-03-31
Project Status Completed (Fiscal Year 2016)
Budget Amount *help
¥4,680,000 (Direct Cost: ¥3,600,000、Indirect Cost: ¥1,080,000)
Fiscal Year 2016: ¥1,430,000 (Direct Cost: ¥1,100,000、Indirect Cost: ¥330,000)
Fiscal Year 2015: ¥1,300,000 (Direct Cost: ¥1,000,000、Indirect Cost: ¥300,000)
Fiscal Year 2014: ¥1,950,000 (Direct Cost: ¥1,500,000、Indirect Cost: ¥450,000)
Keywords先天代謝異常症 / ムコ多糖症 / 新規治療薬 / 骨関節症状 / ポリ硫酸ペントサン / サイトカイン / ウロン酸
Outline of Final Research Achievements

In this study, we investigated the efficacy and safety of pentosan polysulfate in patients with mucopolysaccharidosis about joint symptoms. For adult patients with mucopolysaccharidosis, pentosan polysulfate was injected subcutaneously and safety and therapeutic effects were examined. For 3 adult patients with mucopolysaccharidosis type II, subcutaneous injection of pentosan polysulfate was performed once a week for 12 weeks. After administration of pentosan polysulfate, one patient improved joint pain, and three patients showed mild improvement in the range of joint movement of the shoulder joint. Side reactions were not observed except for mild internal bleeding at the site of subcutaneous injection. Urinary uronate excretion after administration of polysulfate pentosan decreased with one who stopped enzyme replacement therapy. It was considered to act on subcutaneous injection of polysulfate pentosan.

Report

(4 results)
  • 2016 Annual Research Report   Final Research Report ( PDF )
  • 2015 Research-status Report
  • 2014 Research-status Report
  • Research Products

    (3 results)

All 2016 2015

All Presentation (3 results) (of which Int'l Joint Research: 1 results)

  • [Presentation] Safety study of sodium pentosan polysulfate for adult patients with mucopolysaccharidosis type II2016

    • Author(s)
      折居建治、戸松俊治、鈴木康之、Alexander Solyom、Edward H. Schuchman、Calogera M. Simonaro、折居忠夫、深尾敏幸
    • Organizer
      12th Annual WORLD Symposium 2016
    • Place of Presentation
      San Diego(アメリカ合衆国)
    • Year and Date
      2016-02-29
    • Related Report
      2015 Research-status Report
    • Int'l Joint Research
  • [Presentation] ムコ多糖症II型成人患者に対するポリ硫酸ペントサン投与の検討2015

    • Author(s)
      折居建治、戸松俊治、鈴木康之、折居忠夫、深尾敏幸
    • Organizer
      第57回日本先天代謝異常学会
    • Place of Presentation
      大阪市、大阪国際会議場
    • Year and Date
      2015-11-12
    • Related Report
      2015 Research-status Report
  • [Presentation] ムコ多糖症に対するペントサンの臨床研究・臨床治験について2015

    • Author(s)
      折居建治、戸松俊治
    • Organizer
      第19回日本ムコ多糖症研究会
    • Place of Presentation
      大阪市、ホテルアウィーナ大阪
    • Year and Date
      2015-09-21
    • Related Report
      2015 Research-status Report

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Published: 2014-04-04   Modified: 2018-03-22  

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