Patisiran Pharmacokinetics, Pharmacodynamics, and Exposure-Response Analyses in the Phase 3 APOLLO Trial in Patients With Hereditary Transthyretin-Mediated (hATTR) Amyloidosis.

Zhang, Xiaoping; Goel, Varun; Attarwala, Husain; Sweetser, Marianne T; Clausen, Valerie A; Robbie, Gabriel J

Zhang, Xiaoping; Goel, Varun; Attarwala, Husain; Sweetser, Marianne T; Clausen, Valerie A; Robbie, Gabriel J.

Affiliation

Zhang X; Alnylam Pharmaceuticals, Cambridge, MA, USA.
Goel V; Alnylam Pharmaceuticals, Cambridge, MA, USA.
Attarwala H; Alnylam Pharmaceuticals, Cambridge, MA, USA.
Sweetser MT; Alnylam Pharmaceuticals, Cambridge, MA, USA.
Clausen VA; Alnylam Pharmaceuticals, Cambridge, MA, USA.
Robbie GJ; Alnylam Pharmaceuticals, Cambridge, MA, USA.

J Clin Pharmacol ; 60(1): 37-49, 2020 01.

Article in En | MEDLINE | ID: mdl-31322739

Subject(s)

Amyloid Neuropathies, Familial/drug therapy; Prealbumin/antagonists & inhibitors; RNA, Small Interfering/pharmacokinetics; RNA, Small Interfering/therapeutic use; Administration, Intravenous; Aged; Amyloid Neuropathies, Familial/blood; Amyloid Neuropathies, Familial/complications; Dose-Response Relationship, Drug; Double-Blind Method; Female; Humans; Liposomes/administration & dosage; Liposomes/therapeutic use; Liver/metabolism; Male; Middle Aged; Nanoparticles/administration & dosage; Nanoparticles/therapeutic use; Polyneuropathies/drug therapy; Polyneuropathies/etiology; Prealbumin/metabolism; RNA, Small Interfering/administration & dosage; RNAi Therapeutics; Treatment Outcome

Key words

exposure-response; hereditary transthyretin-mediated amyloidosis (hATTR); patisiran; pharmacodynamics (PD); pharmacokinetics (PK); small interfering ribonucleic acid (siRNA)

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Full text: 1 Collection: 01-internacional Database: MEDLINE Main subject: Prealbumin / Amyloid Neuropathies, Familial / RNA, Small Interfering Type of study: Clinical_trials / Etiology_studies Limits: Aged / Female / Humans / Male / Middle aged Language: En Journal: J Clin Pharmacol Year: 2020 Document type: Article Affiliation country: United States

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