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Phase 2 open-label ...
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Adams, David
(författare)
Phase 2 open-label extension study of patisiran, an investigational RNAi therapeutic for the treatment of familial amyloid polyneuropathy
- Artikel/kapitelEngelska2015
Förlag, utgivningsår, omfång ...
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2015-10-14
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BMJ,2015
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printrdacarrier
Nummerbeteckningar
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LIBRIS-ID:oai:DiVA.org:umu-112659
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https://urn.kb.se/resolve?urn=urn:nbn:se:umu:diva-112659URI
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https://doi.org/10.1136/jnnp-2015-312379.27DOI
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Språk:engelska
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Sammanfattning på:engelska
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Ämneskategori:vet swepub-contenttype
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Ämneskategori:art swepub-publicationtype
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Familial amyloid polyneuropathy (FAP) is a progressive disease. Patisiran is an investigational small interfering RNA (siRNA) targeting TTR. The primary objective of the Phase 2 study is to evaluate the safety of 0.3 mg/kg patisiran administered intravenously once every 3 weeks. Twenty-seven patients were enrolled; the mean duration of treatment was 7 months (range 3–12), with 282 doses administered (median of 11 doses/patient). Chronic dosing with patisiran has been generally well tolerated. Two patients experienced serious adverse events regarded as being unrelated to study drug. Infusion-related reactions were observed in 14.8% of the patients, were mild in severity, and did not result in any discontinuations. Sustained TTR lowering of at least 80% was achieved based on serial TTR measurements for over 9 months, with further nadir of up to 89.6% between doses. Neurologic impairment scores were stable after 6 months of treatment with patisiran. A mean decrease from baseline in mNIS+7 of 0.95 points (N=19) observed in this study compared favorably to the estimated increase of 7–10 points in mNIS+7 at 6 months from prior FAP studies in a patient population with similar baseline NIS values. Dosing continues in all patients, and 12–month results will be presented.
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Biuppslag (personer, institutioner, konferenser, titlar ...)
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Suhr, OleUmeå universitet,Medicin(Swepub:umu)olsu0002
(författare)
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Conceicao, Isabel
(författare)
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Waddington-Cruz, Marcia
(författare)
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Schmidt, Hartmut
(författare)
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Buades, Juan
(författare)
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Campistol, Josep
(författare)
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Pouget, Jean
(författare)
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Berk, John
(författare)
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Coelho, Teresa
(författare)
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Umeå universitetMedicin
(creator_code:org_t)
Sammanhörande titlar
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Ingår i:Journal of Neurology, Neurosurgery and Psychiatry: BMJ86:110022-30501468-330X
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Adams, David
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Suhr, Ole
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Conceicao, Isabe ...
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Waddington-Cruz, ...
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Schmidt, Hartmut
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Buades, Juan
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visa fler...
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Campistol, Josep
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Pouget, Jean
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Berk, John
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Coelho, Teresa
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visa färre...
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- MEDICIN OCH HÄLSOVETENSKAP
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