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Phase 2 open-label ...
Phase 2 open-label extension study of patisiran, an investigational RNAi therapeutic for the treatment of familial amyloid polyneuropathy
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Adams, David (författare)
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- Suhr, Ole (författare)
- Umeå universitet,Medicin
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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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(creator_code:org_t)
- 2015-10-14
- 2015
- Engelska.
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Ingår i: Journal of Neurology, Neurosurgery and Psychiatry. - : BMJ. - 0022-3050 .- 1468-330X. ; 86:11
- Relaterad länk:
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https://urn.kb.se/re...
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https://doi.org/10.1...
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Abstract
Ämnesord
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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.
Ämnesord
- MEDICIN OCH HÄLSOVETENSKAP -- Klinisk medicin -- Neurologi (hsv//swe)
- MEDICAL AND HEALTH SCIENCES -- Clinical Medicine -- Neurology (hsv//eng)
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- Av författaren/redakt...
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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...
- Om ämnet
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- MEDICIN OCH HÄLSOVETENSKAP
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MEDICIN OCH HÄLS ...
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och Klinisk medicin
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och Neurologi
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Journal of Neuro ...
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Umeå universitet