CRISPR-engineered T cells in patients with refractory cancer
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Pharmacokinetics and Pharmacodynamics of the Small Interfering Ribonucleic Acid (siRNA), Givosiran, in Patients with Acute Hepatic Porphyria.
Agarwal S, Simon AR, Goel V, Habtemariam BA, Clausen VA, Kim JB, Robbie GJ. Clin Pharmacol Ther. 2020 Jan 29. PubMed
Lipoprotein(a) Reduction in Persons with Cardiovascular Disease.
Tsimikas S, Karwatowska-Prokopczuk E, Gouni-Berthold I, Tardif JC, Baum SJ, Steinhagen-Thiessen E, Shapiro MD, Stroes ES, Moriarty PM, Nordestgaard BG, Xia S, Guerriero J, Viney NJ, O'Dea L, Witztum JL; AKCEA-APO(a)-LRx Study Investigators. N Engl J Med. 2020 Jan 1. PubMed
Analysis of autonomic outcomes in APOLLO, a phase III trial of the RNAi therapeutic patisiran in patients with hereditary transthyretin-mediated amyloidosis.
González-Duarte A, Berk JL, Quan D, Mauermann ML, Schmidt HH, Polydefkis M, Waddington-Cruz M, Ueda M, Conceição IM, Kristen AV, Coelho T, Cauquil CA, Tard C, Merkel M, Aldinc E, Chen J, Sweetser MT, Wang JJ, Adams D. J Neurol. 2019 Nov 14. PubMed ...
Pharmacokinetics of Patisiran, the First Approved RNA Interference Therapy in Patients With Hereditary Transthyretin-Mediated Amyloidosis.
Zhang X, Goel V, Robbie GJ. J Clin Pharmacol. 2019 Nov 27. PubMed
Age-dependent SMN expression in disease-relevant tissue and implications for SMA treatment.
Ramos DM, d'Ydewalle C, Gabbeta V, Dakka A, Klein SK, Norris DA, Matson J, Taylor SJ, Zaworski PG, Prior TW, Snyder PJ, Valdivia D, Hatem CL, Waters I, Gupte N, Swoboda KJ, Rigo F, Bennett CF, Naryshkin N, Paushkin S, Crawford TO, Sumner CJ. J ...
Nusinersen initiated in infants during the presymptomatic stage of spinal muscular atrophy: Interim efficacy and safety results from the Phase 2 NURTURE study.
De Vivo DC, Bertini E, Swoboda KJ, Hwu WL, Crawford TO, Finkel RS, Kirschner J, Kuntz NL, Parsons JA, Ryan MM, Butterfield RJ, Topaloglu H, Ben-Omran T, Sansone VA, Jong YJ, Shu F, Staropoli JF, Kerr D, Sandrock AW, Stebbins C, Petrillo M, Braley G, ...
RNA Interference Therapy with ARC-520 Results in Prolonged HBsAg Response in Patients with Chronic Hepatitis B Infection.
Yuen MF, Schiefke I, Yoon JH, Ahn SH, Heo J, Kim JH, Chan HLY, Yoon KT, Klinker H, Manns M, Petersen J, Schluep T, Hamilton J, Given BD, Ferrari C, Lai CL, Locarnini SA, Gish RG. Hepatology. 2019 Oct 26. PubMed
Patient-Customized Oligonucleotide Therapy for a Rare Genetic Disease
Kim J, Hu C, Moufawad El Achkar C, Black LE, Douville J, Larson A, Pendergast MK, Goldkind SF, Lee EA, Kuniholm A, Soucy A, Vaze J, Belur NR, Fredriksen K, Stojkovska I, Tsytsykova A, Armant M, DiDonato RL, Choi J, Cornelissen L, Pereira LM, Augustine ...
Therapy with 2′-O-Me Phosphorothioate Antisense Oligonucleotides Causes Reversible Proteinuria by Inhibiting Renal Protein Reabsorption
Janssen MJ, Nieskens TTG, Steevels TAM, Caetano-Pinto P, den Braanker D, Mulder M, Ponstein Y, Jones S, Masereeuw R, den Besten C, Wilmer MJ. Mol Ther Nucleic Acids. 2019 Sep 6;18:298-307. PubMed
