2013 Fiscal Year Final Research Report
Establishment of early diagnosis method and new non-invasive therapy for transthyretin amyloidosis
| Project/Area Number |
23591237
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| Research Category |
Grant-in-Aid for Scientific Research (C)
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| Allocation Type | Multi-year Fund |
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| Section | 一般 |
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| Research Field |
Neurology
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| Research Institution | Shinshu University |
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Principal Investigator |
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| Co-Investigator(Kenkyū-buntansha) |
TOJO Kana 信州大学, 医学部附属病院, 助教(特定雇用) (10467178)
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| Co-Investigator(Renkei-kenkyūsha) |
MORITA Hiroshi 信州大学, 医学部附属病院, 准教授 (10262718)
KOYAMA Jun 信州大学, 医学系研究科, 准教授 (10303463)
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| Project Period (FY) |
2011 – 2013
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| Keywords | アミロイド / トランスサイレチン / 老化 / 蛋白質 / 早期診断 / 新規治療 |
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| Research Abstract |
We showed that abdominal fat biopsy is the most sensitive early diagnosis method for familial amyloid polyneuropathy (FAP). We also showed that endomyocardial biopsy or gastroduodenal biopsy is the most sensitive early diagnosis method for senile systemic amyloidosis (SSA). Surgical skin biopsy including the deep subcutaneous fat pad is a useful alternative histopathological diagnosis tool for SSA when patient is in poor condition.
With regard to treatment, we demonstrated that diflunisal inhibit polyneuropathy progression and preserve quality of life in patients with FAP in its 2-year randomized controlled trial. We also analyzed the tolerance and efficacy of long-term diflunisal administration in FAP patients and showed that clinical effects of diflunisal are sustained after two years of treatment.
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Research Products
(53 results)
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[Journal Article] Repurposing diflunisal for familial amyloid polyneuropathy : a randomized clinical trial2013
Author(s)
Berk JL, Suhr OB, Obici L, Sekijima Y, Zeldenrust SR, Yamashita T, Heneghan MA, Gorevic PD, Litchy WJ, Wiesman JF, Nordh E, Corato M, Lozza A, Cortese A, Robinson-Papp J, Colton T, Rybin DV, Bisbee AB, Ando Y, Ikeda S, Seldin DC, Merlini G, Skinner M, Kelly JW, Dyck PJ; Diflunisal Trial Consortium
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Journal Title
JAMA
Volume: 310
Pages: 2658-2667
DOI
Peer Reviewed
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[Journal Article] Two Siblings Diagnosed to Have Transthyretin-related Familial Amyloid Cardiomyopathy Around the Same Time at Different Hospitals2012
Author(s)
Miyamura M, Terasaki F, Ishibashi K, Shimazaki C, Kimura F, Kuwabara H, Tsuji M, Shibayama Y, Sekijima Y, Tojo K, Ishizaka N
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Journal Title
Intern Med
Volume: 51
Pages: 465-9
Peer Reviewed
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[Journal Article] The Diflunisal Trial : study accrual and drug tolerance2012
Author(s)
Berk JL, Suhr OB, Sekijima Y, Yamashita T, Heneghan M, Zeldenrust SR, Ando Y, Ikeda S, Gorevic P, Merlini G, Kelly JW, Skinner M, Bisbee AB, Dyck PJ, Obici L; Familial Amyloidosis Consortium
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Journal Title
Amyloid
Volume: 19 Suppl 1
Pages: 37-8
DOI
Peer Reviewed
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[Presentation] The Diflunisal Trial : Demographics,baseline neurologic sraging, and adverse events2012
Author(s)
Berk JL, Obici L, Zeldenrust SR, Sekijima Y, Yamashita T, Heneghan M, Ikeda S, Ando Y,Gorevic P, Merlini G, Kelly JW, Skinner M, Bisbee AB, Dyck PJ, Suhr OR, Familial Amyloidosis Consortium
Organizer
XIII^<th> International Symposium on Amyloidosis
Place of Presentation
Groningen, Netherland
Year and Date
20120506-10
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[Presentation] The Diflunisal Trial : Study accrual and drug tolerance2011
Author(s)
Berk JL, Suhr OB, Sekijima Y, Yamashita T, Heneghan M, Zeldenrust SR, Ando Y, Ikeda S-I, Gorevic P, Merlini G, Kelly JW, Skinner M, Bisbee AB, Dyck PJ, Obici L, et al
Organizer
VIIIth International Symposium on Familial Amyloidotic Polyneuropathy
Place of Presentation
Kumamoto
Year and Date
20111120-22
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