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265 results

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Page 1
Patisiran, an RNAi Therapeutic, for Hereditary Transthyretin Amyloidosis.
Adams D, Gonzalez-Duarte A, O'Riordan WD, Yang CC, Ueda M, Kristen AV, Tournev I, Schmidt HH, Coelho T, Berk JL, Lin KP, Vita G, Attarian S, Planté-Bordeneuve V, Mezei MM, Campistol JM, Buades J, Brannagan TH 3rd, Kim BJ, Oh J, Parman Y, Sekijima Y, Hawkins PN, Solomon SD, Polydefkis M, Dyck PJ, Gandhi PJ, Goyal S, Chen J, Strahs AL, Nochur SV, Sweetser MT, Garg PP, Vaishnaw AK, Gollob JA, Suhr OB. Adams D, et al. N Engl J Med. 2018 Jul 5;379(1):11-21. doi: 10.1056/NEJMoa1716153. N Engl J Med. 2018. PMID: 29972753 Free article. Clinical Trial.
BACKGROUND: Patisiran, an investigational RNA interference therapeutic agent, specifically inhibits hepatic synthesis of transthyretin. ...CONCLUSIONS: In this trial, patisiran improved multiple clinical manifestations of hereditary transthyretin amyloidosis. ...
BACKGROUND: Patisiran, an investigational RNA interference therapeutic agent, specifically inhibits hepatic synthesis of transthyreti …
Patisiran: First Global Approval.
Hoy SM. Hoy SM. Drugs. 2018 Oct;78(15):1625-1631. doi: 10.1007/s40265-018-0983-6. Drugs. 2018. PMID: 30251172 Review.
Patisiran (ONPATTRO) is a double-stranded small interfering RNA encapsulated in a lipid nanoparticle for delivery to hepatocytes. ...This article summarizes the milestones in the development of patisiran leading to these approvals....
Patisiran (ONPATTRO) is a double-stranded small interfering RNA encapsulated in a lipid nanoparticle for delivery to hepatocytes. ... …
Patisiran for the Treatment of Transthyretin-mediated Amyloidosis with Cardiomyopathy.
Ioannou A, Fontana M, Gillmore JD. Ioannou A, et al. Heart Int. 2023 Jun 8;17(1):27-35. doi: 10.17925/HI.2023.17.1.27. eCollection 2023. Heart Int. 2023. PMID: 37456349 Free PMC article. Review.
After encouraging results following the use of patisiran for the treatment of patients with ATTR-PN, there has been increasing interest in the use of patisiran for the treatment of ATTR-CM. ...These successes represent a huge milestone and have the potential to revo …
After encouraging results following the use of patisiran for the treatment of patients with ATTR-PN, there has been increasing intere …
Patisiran.
[No authors listed] [No authors listed] 2023 Jan 6. LiverTox: Clinical and Research Information on Drug-Induced Liver Injury [Internet]. Bethesda (MD): National Institute of Diabetes and Digestive and Kidney Diseases; 2012–. 2023 Jan 6. LiverTox: Clinical and Research Information on Drug-Induced Liver Injury [Internet]. Bethesda (MD): National Institute of Diabetes and Digestive and Kidney Diseases; 2012–. PMID: 36689610 Free Books & Documents. Review.
Patisiran is a synthetic small interfering RNA (siRNA) molecule directed against the mRNA of transthyretin that is used to treat the rare genetic disease transthyretin-mediated amyloidosis. Patisiran has not been linked to serum aminotransferase elevations during th …
Patisiran is a synthetic small interfering RNA (siRNA) molecule directed against the mRNA of transthyretin that is used to treat the …
Patisiran Treatment in Patients with Transthyretin Cardiac Amyloidosis.
Maurer MS, Kale P, Fontana M, Berk JL, Grogan M, Gustafsson F, Hung RR, Gottlieb RL, Damy T, González-Duarte A, Sarswat N, Sekijima Y, Tahara N, Taylor MS, Kubanek M, Donal E, Palecek T, Tsujita K, Tang WHW, Yu WC, Obici L, Simões M, Fernandes F, Poulsen SH, Diemberger I, Perfetto F, Solomon SD, Di Carli M, Badri P, White MT, Chen J, Yureneva E, Sweetser MT, Jay PY, Garg PP, Vest J, Gillmore JD; APOLLO-B Trial Investigators. Maurer MS, et al. N Engl J Med. 2023 Oct 26;389(17):1553-1565. doi: 10.1056/NEJMoa2300757. N Engl J Med. 2023. PMID: 37888916 Free PMC article. Clinical Trial.
RESULTS: A total of 360 patients were randomly assigned to receive patisiran (181 patients) or placebo (179 patients). At month 12, the decline in the 6-minute walk distance was lower in the patisiran group than in the placebo group (Hodges-Lehmann estimate of media …
RESULTS: A total of 360 patients were randomly assigned to receive patisiran (181 patients) or placebo (179 patients). At month 12, t …
Effects of Patisiran, an RNA Interference Therapeutic, on Cardiac Parameters in Patients With Hereditary Transthyretin-Mediated Amyloidosis.
Solomon SD, Adams D, Kristen A, Grogan M, González-Duarte A, Maurer MS, Merlini G, Damy T, Slama MS, Brannagan TH 3rd, Dispenzieri A, Berk JL, Shah AM, Garg P, Vaishnaw A, Karsten V, Chen J, Gollob J, Vest J, Suhr O. Solomon SD, et al. Circulation. 2019 Jan 22;139(4):431-443. doi: 10.1161/CIRCULATIONAHA.118.035831. Circulation. 2019. PMID: 30586695 Clinical Trial.
The APOLLO study assessed the efficacy and tolerability of patisiran in patients with hATTR amyloidosis. The effects of patisiran on cardiac structure and function in a prespecified subpopulation of patients with evidence of cardiac amyloid involvement at baseline w …
The APOLLO study assessed the efficacy and tolerability of patisiran in patients with hATTR amyloidosis. The effects of patisiran …
Long-term safety and efficacy of patisiran for hereditary transthyretin-mediated amyloidosis with polyneuropathy: 12-month results of an open-label extension study.
Adams D, Polydefkis M, González-Duarte A, Wixner J, Kristen AV, Schmidt HH, Berk JL, Losada López IA, Dispenzieri A, Quan D, Conceição IM, Slama MS, Gillmore JD, Kyriakides T, Ajroud-Driss S, Waddington-Cruz M, Mezei MM, Planté-Bordeneuve V, Attarian S, Mauricio E, Brannagan TH 3rd, Ueda M, Aldinc E, Wang JJ, White MT, Vest J, Berber E, Sweetser MT, Coelho T; patisiran Global OLE study group. Adams D, et al. Lancet Neurol. 2021 Jan;20(1):49-59. doi: 10.1016/S1474-4422(20)30368-9. Epub 2020 Nov 16. Lancet Neurol. 2021. PMID: 33212063 Clinical Trial.
At 12 months, improvements in mNIS+7 with patisiran were sustained from parent study baseline with treatment in the global OLE (APOLLO-patisiran mean change -4.0, 95 % CI -7.7 to -0.3; phase 2 OLE patisiran -4.7, -11.9 to 2.4). ...Serious adverse events were …
At 12 months, improvements in mNIS+7 with patisiran were sustained from parent study baseline with treatment in the global OLE (APOLL …
Patisiran for the treatment of hereditary transthyretin-mediated amyloidosis.
Yang J. Yang J. Expert Rev Clin Pharmacol. 2019 Feb;12(2):95-99. doi: 10.1080/17512433.2019.1567326. Epub 2019 Jan 18. Expert Rev Clin Pharmacol. 2019. PMID: 30644768 Review.
Areas covered: In this review, the chemical property, mechanism of action, pharmacokinetics, clinical efficacy, and safety of patisiran were introduced. Expert Commentary: Patisiran offers a new treatment option for patients with hereditary transthyretin-mediated am …
Areas covered: In this review, the chemical property, mechanism of action, pharmacokinetics, clinical efficacy, and safety of patisiran …
Patisiran.
Kalola UK, Pellegrini MV. Kalola UK, et al. 2023 Mar 17. In: StatPearls [Internet]. Treasure Island (FL): StatPearls Publishing; 2024 Jan–. 2023 Mar 17. In: StatPearls [Internet]. Treasure Island (FL): StatPearls Publishing; 2024 Jan–. PMID: 36972364 Free Books & Documents.
Patisiran is indicated for treating polyneuropathy associated with hereditary transthyretin-mediated amyloidosis (hATTR) in adults. ...
Patisiran is indicated for treating polyneuropathy associated with hereditary transthyretin-mediated amyloidosis (hATTR) in adults. . …
Patisiran Pharmacokinetics, Pharmacodynamics, and Exposure-Response Analyses in the Phase 3 APOLLO Trial in Patients With Hereditary Transthyretin-Mediated (hATTR) Amyloidosis.
Zhang X, Goel V, Attarwala H, Sweetser MT, Clausen VA, Robbie GJ. Zhang X, et al. J Clin Pharmacol. 2020 Jan;60(1):37-49. doi: 10.1002/jcph.1480. Epub 2019 Jul 19. J Clin Pharmacol. 2020. PMID: 31322739 Free PMC article. Clinical Trial.
The lipid nanoparticle also contains 2 novel lipid excipients (DLin-MC3-DMA and PEG(2000) -C-DMG). Here we report patisiran pharmacokinetics (PK), pharmacodynamics (PD), and exposure-response analyses from the phase 3 APOLLO trial, in which patients with hATTR amyloidosis …
The lipid nanoparticle also contains 2 novel lipid excipients (DLin-MC3-DMA and PEG(2000) -C-DMG). Here we report patisiran pharmacok …
265 results