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The diflunisal trial: study accrual and drug tolerance
Umeå University, Faculty of Medicine, Department of Public Health and Clinical Medicine, Medicine.
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2012 (English)In: Amyloid: Journal of Protein Folding Disorders, ISSN 1350-6129, E-ISSN 1744-2818, Vol. 19, no S1, 37-38 p.Article in journal (Refereed) Published
Abstract [en]

Familial amyloidotic polyneuropathy (FAP) is a protein folding disorder that induces neuropathy and cardiomyopathy, leading to death within 7-15 years after onset of clinical disease. In vitro, small ligands binding the thyroid hormone docking site stabilize tetrameric transthyretin, inhibiting amyloid fibril formation. We undertook a randomized, placebo-controlled clinical trial to determine whether diflunisal, a well-known non-steroidal anti-inflammatory drug (NSAID) alters neurologic disease progression in FAP. We enrolled 130 subjects with wide age and FAP mutation representation. To date, few recognized complications of NSAIDs have occurred in the study cohort. Data collection will be completed by November 2012.

Place, publisher, year, edition, pages
2012. Vol. 19, no S1, 37-38 p.
Keyword [en]
Clinical trials, diflunisal, FAP, neuropathy, transthyretin
National Category
Family Medicine
Identifiers
URN: urn:nbn:se:umu:diva-56404DOI: 10.3109/13506129.2012.678509ISI: 000304406400013OAI: oai:DiVA.org:umu-56404DiVA: diva2:535589
Available from: 2012-06-20 Created: 2012-06-18 Last updated: 2017-12-07Bibliographically approved

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