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49 results

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Page 1
A Highly Durable RNAi Therapeutic Inhibitor of PCSK9.
Fitzgerald K, White S, Borodovsky A, Bettencourt BR, Strahs A, Clausen V, Wijngaard P, Horton JD, Taubel J, Brooks A, Fernando C, Kauffman RS, Kallend D, Vaishnaw A, Simon A. Fitzgerald K, et al. Among authors: vaishnaw a. N Engl J Med. 2017 Jan 5;376(1):41-51. doi: 10.1056/NEJMoa1609243. Epub 2016 Nov 13. N Engl J Med. 2017. PMID: 27959715 Free PMC article. Clinical Trial.
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. Among authors: vaishnaw ak. 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.
Lumasiran, an RNAi Therapeutic for Primary Hyperoxaluria Type 1.
Garrelfs SF, Frishberg Y, Hulton SA, Koren MJ, O'Riordan WD, Cochat P, Deschênes G, Shasha-Lavsky H, Saland JM, Van't Hoff WG, Fuster DG, Magen D, Moochhala SH, Schalk G, Simkova E, Groothoff JW, Sas DJ, Meliambro KA, Lu J, Sweetser MT, Garg PP, Vaishnaw AK, Gansner JM, McGregor TL, Lieske JC; ILLUMINATE-A Collaborators. Garrelfs SF, et al. Among authors: vaishnaw ak. N Engl J Med. 2021 Apr 1;384(13):1216-1226. doi: 10.1056/NEJMoa2021712. N Engl J Med. 2021. PMID: 33789010 Clinical Trial.
Rare loss of function variants in the hepatokine gene INHBE protect from abdominal obesity.
Deaton AM, Dubey A, Ward LD, Dornbos P, Flannick J; AMP-T2D-GENES Consortium; Yee E, Ticau S, Noetzli L, Parker MM, Hoffing RA, Willis C, Plekan ME, Holleman AM, Hinkle G, Fitzgerald K, Vaishnaw AK, Nioi P. Deaton AM, et al. Among authors: vaishnaw ak. Nat Commun. 2022 Jul 27;13(1):4319. doi: 10.1038/s41467-022-31757-8. Nat Commun. 2022. PMID: 35896531 Free PMC article.
RNA interference in the era of nucleic acid therapeutics.
Jadhav V, Vaishnaw A, Fitzgerald K, Maier MA. Jadhav V, et al. Among authors: vaishnaw a. Nat Biotechnol. 2024 Mar;42(3):394-405. doi: 10.1038/s41587-023-02105-y. Epub 2024 Feb 26. Nat Biotechnol. 2024. PMID: 38409587 Review.
The Nonclinical Disposition and Pharmacokinetic/Pharmacodynamic Properties of N-Acetylgalactosamine-Conjugated Small Interfering RNA Are Highly Predictable and Build Confidence in Translation to Human.
McDougall R, Ramsden D, Agarwal S, Agarwal S, Aluri K, Arciprete M, Brown C, Castellanos-Rizaldos E, Charisse K, Chong S, Cichocki J, Fitzgerald K, Goel V, Gu Y, Guenther D, Habtemariam B, Jadhav V, Janas M, Jayaraman M, Kurz J, Li J, Liu J, Liu X, Liou S, Maclauchlin C, Maier M, Manoharan M, Nair JK, Robbie G, Schmidt K, Smith P, Theile C, Vaishnaw A, Waldron S, Xu Y, Zhang X, Zlatev I, Wu JT. McDougall R, et al. Among authors: vaishnaw a. Drug Metab Dispos. 2022 Jun;50(6):781-797. doi: 10.1124/dmd.121.000428. Epub 2021 Jun 21. Drug Metab Dispos. 2022. PMID: 34154993 Review.
Safety and efficacy of RNAi therapy for transthyretin amyloidosis.
Coelho T, Adams D, Silva A, Lozeron P, Hawkins PN, Mant T, Perez J, Chiesa J, Warrington S, Tranter E, Munisamy M, Falzone R, Harrop J, Cehelsky J, Bettencourt BR, Geissler M, Butler JS, Sehgal A, Meyers RE, Chen Q, Borland T, Hutabarat RM, Clausen VA, Alvarez R, Fitzgerald K, Gamba-Vitalo C, Nochur SV, Vaishnaw AK, Sah DW, Gollob JA, Suhr OB. Coelho T, et al. Among authors: vaishnaw ak. N Engl J Med. 2013 Aug 29;369(9):819-29. doi: 10.1056/NEJMoa1208760. N Engl J Med. 2013. PMID: 23984729 Free article. Clinical Trial.
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. Among authors: vaishnaw a. Circulation. 2019 Jan 22;139(4):431-443. doi: 10.1161/CIRCULATIONAHA.118.035831. Circulation. 2019. PMID: 30586695 Clinical Trial.
Single-Dose Pharmacokinetics and Pharmacodynamics of Transthyretin Targeting N-acetylgalactosamine-Small Interfering Ribonucleic Acid Conjugate, Vutrisiran, in Healthy Subjects.
Habtemariam BA, Karsten V, Attarwala H, Goel V, Melch M, Clausen VA, Garg P, Vaishnaw AK, Sweetser MT, Robbie GJ, Vest J. Habtemariam BA, et al. Among authors: vaishnaw ak. Clin Pharmacol Ther. 2021 Feb;109(2):372-382. doi: 10.1002/cpt.1974. Epub 2020 Aug 13. Clin Pharmacol Ther. 2021. PMID: 32599652 Clinical Trial.
Phase 3 Trial of RNAi Therapeutic Givosiran for Acute Intermittent Porphyria.
Balwani M, Sardh E, Ventura P, Peiró PA, Rees DC, Stölzel U, Bissell DM, Bonkovsky HL, Windyga J, Anderson KE, Parker C, Silver SM, Keel SB, Wang JD, Stein PE, Harper P, Vassiliou D, Wang B, Phillips J, Ivanova A, Langendonk JG, Kauppinen R, Minder E, Horie Y, Penz C, Chen J, Liu S, Ko JJ, Sweetser MT, Garg P, Vaishnaw A, Kim JB, Simon AR, Gouya L; ENVISION Investigators. Balwani M, et al. Among authors: vaishnaw a. N Engl J Med. 2020 Jun 11;382(24):2289-2301. doi: 10.1056/NEJMoa1913147. N Engl J Med. 2020. PMID: 32521132 Clinical Trial.
49 results