Immunogenicity and safety of a 2-dose hepatitis B vaccine, HBsAg/CpG 1018, in persons with diabetes mellitus aged 60-70 years
Hyer RN, Janssen RS. Vaccine. 2019 Sep 16;37(39):5854-5861. PubMed
Volanesorsen and Triglyceride Levels in Familial Chylomicronemia Syndrome
Witztum JL, Gaudet D, Freedman SD, Alexander VJ, Digenio A, Williams KR, Yang Q, Hughes SG, Geary RS, Arca M, Stroes ESG, Bergeron J, Soran H, Civeira F, Hemphill L, Tsimikas S, Blom DJ, O'Dea L, Bruckert E. N Engl J Med. 2019 Aug 8;381(6):531-542. ...
Metabolism and Disposition of Volanesorsen, a 2′ MOE Antisense Oligonucleotide, Across Species
Post N, Yu R, Greenlee S, Gaus H, Hurh E, Matson J, Wang Y. Drug Metab Dispos. 2019 Jul 26. PubMed
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. J Clin Pharmacol. 2019 Jul 19. PubMed
Adoptive Transfer of NKG2D CAR mRNA-Engineered Natural Killer Cells in Colorectal Cancer Patients
Xiao L, Cen D, Gan H, Sun Y, Huang N, Xiong H, Jin Q, Su L, Liu X, Wang K, Yan G, Dong T, Wu S, Zhou P, Zhang J, Liang W, Ren J, Teng Y, Chen C, Xu XH. Mol Ther. 2019 Jun 5;27(6):1114-1125. ...
Targeting Huntingtin Expression in Patients with Huntington’s Disease
Tabrizi SJ, Leavitt BR, Landwehrmeyer GB, Wild EJ, Saft C, Barker RA, Blair NF, Craufurd D, Priller J, Rickards H, Rosser A, Kordasiewicz HB, Czech C, Swayze EE, Norris DA, Baumann T, Gerlach I, Schobel SA, Paz E, Smith AV, Bennett CF, Lane RM. N ...
Nusinersen in later-onset spinal muscular atrophy: Long-term results from the phase 1/2 studies
Darras BT, Chiriboga CA, Iannaccone ST, Swoboda KJ, Montes J, Mignon L, Xia S, Bennett CF, Bishop KM, Shefner JM, Green AM, Sun P, Bhan I, Gheuens S, Schneider E, Farwell W, De Vivo DC; ISIS-396443-CS2/ISIS-396443-CS12 Study Groups; ISIS-396443-CS2/ISIS-396443-CS12 Study. Neurology. 2019 Apr 24. PubMed
Association of Patisiran, an RNA Interference Therapeutic, With Regional Left Ventricular Myocardial Strain in Hereditary Transthyretin Amyloidosis: The APOLLO Study
Minamisawa M, Claggett B, Adams D, Kristen AV, Merlini G, Slama MS, Dispenzieri A, Shah AM, Falk RH, Karsten V, Sweetser MT, Chen J, Riese R, Vest J, Solomon SD. JAMA Cardiol. 2019 Mar 16. PubMed
IONIS-PKKRx a Novel Antisense Inhibitor of Prekallikrein and Bradykinin Production
Ferrone JD, Bhattacharjee G, Revenko AS, Zanardi TA, Warren MS, Derosier FJ, Viney NJ, Pham NC, Kaeser GE, Baker BF, Schneider E, Hughes SG, Monia BP, MacLeod AR. Nucleic Acid Ther. 2019 Feb 28. PubMed
Phase 1 Trial of an RNA Interference Therapy for Acute Intermittent Porphyria
Sardh E, Harper P, Balwani M, Stein P, Rees D, Bissell DM, Desnick R, Parker C, Phillips J, Bonkovsky HL, Vassiliou D, Penz C, Chan-Daniels A, He Q, Querbes W, Fitzgerald K, Kim JB, Garg P, Vaishnaw A, Simon AR, Anderson KE. N Engl J ...
