Publication Information

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'15': [23, 104, 1002], 'years': [24, 105, 1116, 2079, 2972], 'ago': [25, 106], 'by': [26, 107, 169, 305, 365, 371, 448, 454, 465, 509, 515, 680, 724, 757, 937, 1163, 1169, 1188, 1211, 1221, 1230, 1243, 1346, 1472, 1643, 1679, 1786, 2451, 2541, 2628, 2749, 2765, 2910, 3170, 3452, 3469, 3546, 4075, 4312], 'siRNAs.': [27, 108], 'During': [28, 109], 'that': [29, 61, 110, 142, 218, 884, 952, 998, 2661, 2734, 2756, 2797, 2818, 3052, 3147, 3207, 3568, 4183, 4253], 'lengthy': [30, 111], 'period': [31, 112], 'of': [32, 42, 54, 73, 113, 123, 135, 154, 184, 207, 245, 250, 285, 290, 293, 296, 313, 346, 353, 358, 378, 402, 409, 452, 463, 487, 556, 579, 616, 623, 667, 677, 733, 742, 749, 761, 765, 774, 785, 798, 890, 904, 910, 928, 940, 967, 977, 983, 1004, 1021, 1027, 1048, 1075, 1084, 1087, 1117, 1234, 1255, 1278, 1291, 1307, 1337, 1348, 1362, 1368, 1371, 1373, 1407, 1438, 1482, 1529, 1539, 1609, 1625, 1638, 1676, 1687, 1699, 1737, 1780, 1803, 1810, 1819, 1835, 1883, 1930, 1980, 2060, 2067, 2080, 2117, 2169, 2206, 2238, 2335, 2410, 2458, 2504, 2513, 2528, 2544, 2559, 2619, 2640, 2649, 2678, 2684, 2708, 2717, 2760, 2790, 2800, 2823, 2845, 2857, 2875, 2879, 2886, 2890, 2918, 2926, 3020, 3039, 3088, 3154, 3190, 3198, 3214, 3234, 3263, 3310, 3333, 3358, 3503, 3533, 3536, 3559, 3597, 3635, 3640, 3713, 3719, 3721, 3744, 3756, 3789, 3913, 3956, 4019, 4172, 4179, 4231, 4240, 4260, 4272, 4278, 4294, 4306], 'time,': [33, 114, 385], 'numerous': [34, 115, 3746, 4295], 'trials': [36, 117, 807, 1180, 1852, 2589], 'performed': [39, 120, 1177], 'thousands': [41, 122], 'trial': [43, 124, 244, 1929, 1985, 2062, 2145, 2168, 2190, 2243, 2267, 2429, 2524, 2601, 4057, 4181], 'participants': [44, 125], 'accrued': [45, 126], 'onto': [46, 127], 'studies.': [47, 128], 'Of': [48, 129], 'all': [49, 130, 578, 591, 4156], 'molecules': [51, 132], 'evaluated': [52, 133, 801, 2056, 3116], 'as': [53, 134, 821, 1290, 1991, 2249, 2403, 2453, 2774, 3126, 3173, 3286, 3441, 3812, 4087, 4169], 'January': [55, 136, 2834], '2017,': [56, 137, 1292], 'regulatory': [58, 139, 2682, 3660, 4076], 'authorities': [59, 140], 'assessed': [60, 141], 'six': [62, 75, 143, 156], 'provided': [63, 144], 'clear': [64, 145], 'benefit': [66, 147, 1133], 'in': [67, 78, 148, 159, 181, 256, 351, 368, 381, 419, 421, 424, 426, 488, 564, 700, 744, 753, 782, 791, 802, 838, 865, 957, 1016, 1042, 1058, 1092, 1101, 1135, 1138, 1153, 1173, 1192, 1207, 1214, 1228, 1252, 1312, 1318, 1351, 1357, 1387, 1442, 1576, 1777, 1798, 1837, 1887, 1932, 1975, 1994, 2029, 2057, 2086, 2121, 2171, 2233, 2252, 2298, 2382, 2389, 2396, 2414, 2461, 2471, 2495, 2499, 2521, 2546, 2561, 2586, 2600, 2603, 2645, 2654, 2667, 2740, 2745, 2809, 2854, 3117, 3136, 3180, 3304, 3361, 3398, 3447, 3475, 3510, 3529, 3556, 3572, 3605, 3691, 3701, 3747, 3750, 3856, 3920, 3964, 4026, 4041, 4055, 4199, 4212, 4222, 4263], 'rigorously': [68, 149], 'controlled': [69, 150, 242], 'trials.': [70, 151, 805, 960], 'The': [71, 152, 162, 213, 316, 443, 485, 566, 794, 892, 1112, 1216, 1536, 1615, 2376, 2731, 2781, 2814, 2948, 3031, 3102, 3307, 3318, 3460, 3553, 3717, 4050, 4174], 'story': [72, 153], 'these': [74, 155, 1088, 1096, 1738, 3311], 'is': [76, 157, 226, 433, 561, 738, 779, 953, 1218, 1286, 1310, 1328, 1344, 1543, 1683, 1732, 1790, 2928, 2933, 2996, 3065, 3082, 3134, 3168, 3321, 3458, 3463, 3524, 3564, 3689, 4066], 'given': [77, 158], 'this': [79, 160, 1377, 2919, 3547, 4056, 4180], 'review.': [80, 161], 'first': [163, 446, 2967], 'oligonucleotide': [165, 1572, 1627], 'approved': [166, 364, 723, 1037, 1311, 2943, 3097], 'marketing': [168, 416, 2699, 2844], 'Food': [171], 'Drug': [173, 541], 'Administration': [174], '(FDA)': [175], 'was': [176, 203, 215, 276, 329, 363, 387, 445, 653, 722, 800, 894, 935, 1108, 1166, 1186, 1209, 1226, 2055, 2386, 2449, 2497, 2539, 2643, 2686, 2783, 2808, 2860, 4039, 4058, 4194], 'fomivirsen,': [177], 'a': [178, 182, 196, 232, 388, 532, 552, 569, 715, 931, 950, 1129, 1158, 1296, 1329, 1393, 1448, 1775, 1893, 1981, 2038, 2127, 2204, 2239, 2307, 2421, 2488, 2518, 2542, 2596, 2636, 2757, 2866, 2934, 2951, 3021, 3028, 3105, 3609, 3678, 3699, 3968, 3990, 4313], 'drug': [179, 418, 893, 1139, 1199, 1217, 1263, 2736, 2764, 2849, 2900, 4146, 4162], 'developed': [180, 1162], 'collaboration': [183, 1174, 2889], 'Isis': [185, 2891], 'Pharmaecuticals': [186], 'Novartis': [188, 414], 'Ophthalmics.': [189], 'This': [190, 736, 1262, 1327, 1355, 1667, 1771, 2859, 3131, 3444, 3676, 3695, 4064], '21-mer': [191], 'phosphorothioate': [192, 570, 1600, 2961], 'oligodeoxynucleotide': [193], '(which': [194], 'contains': [195, 568], 'CpG': [197], 'motif': [198], 'near': [199], 'its': [200, 706, 1741, 2829, 3334, 3339, 3366], '5′': [201, 657], 'terminus)': [202], 'intended': [204], 'treatment': [206, 249, 889, 1119, 1306, 1481, 1698, 2807, 3153, 3213, 3262, 3788, 4285], 'patients': [208, 257, 883, 929, 941, 968, 984, 1089, 1143, 1375, 1443, 1888, 1933, 1995, 2030, 2122, 2172, 2253, 2299, 2326, 2343, 2353, 2369, 2432, 2444, 2466, 2509, 2516, 2531, 2550, 2604, 2614, 2622, 2741, 2761, 2880, 3120, 3399, 3473, 3538, 3561, 3714, 3857, 4043, 4109, 4142, 4158, 4215, 4234], 'cytomegalovirus': [210, 254], '(CMV)': [211], 'retinitis.': [212, 287], 'target': [214], 'mRNA': [217, 1612, 1678, 1754, 3193, 3229], 'encoded': [219], 'CMV': [221, 286, 410], 'immediate-early': [222], '(IE)-2': [223], 'protein,': [224, 3685], 'which': [225, 1172, 1206, 1225, 1285, 2869, 2965, 3025, 3702, 4042], 'required': [227, 2064, 2147, 2192, 2269], 'viral': [229], 'replication.': [230], 'In': [231, 1182, 1547, 1815, 2323, 2428, 2441, 2523, 2713, 3988, 4266], 'study': [233, 864, 1013], 'from': [234, 1200, 1238, 1258, 1274, 1487, 1704, 2380, 2865, 3435, 4206], 'Vitravene': [236, 362], 'Study': [237, 239, 837, 3258, 3494, 3784, 3904, 4010], 'Group,1Vitravene': [238], 'GroupA': [240], 'randomized': [241, 1179, 2329, 2530], 'intravitreous': [246], 'fomivirsen': [247, 275, 297], 'newly': [251], 'diagnosed': [252], 'peripheral': [253], 'retinitis': [255, 314, 395], 'AIDS.Am.': [259], 'J.': [260, 593, 597, 850, 1474, 1691, 3255, 3389, 3488, 3781, 3847, 3898, 4004], 'Ophthalmol.': [261, 873], '2002;': [262], '133:': [263], '467-474Abstract': [264], 'Full': [265, 267, 1456, 1458, 1504, 1506, 1721, 1723, 1901, 1903, 2135, 2137, 3419, 3421, 3619, 3621, 3877, 3879, 3978, 3980], 'Text': [266, 268, 1457, 1459, 1505, 1507, 1722, 1724, 1902, 1904, 2136, 2138, 3420, 3422, 3620, 3622, 3878, 3880, 3979, 3981], 'PDF': [269, 1460, 1508, 1725, 1905, 2139, 3423, 3623, 3881, 3982], 'PubMed': [270, 478, 504, 546, 642, 692, 855, 1461, 1509, 1588, 1726, 1766, 1906, 1946, 2003, 2049, 2140, 2185, 2261, 2318, 3162, 3222, 3271, 3424, 3518, 3624, 3671, 3797, 3882, 3928, 3983, 4034], 'Scopus': [271, 479, 547, 643, 693, 856, 1462, 1510, 1589, 1663, 1727, 1767, 1907, 1947, 2004, 2050, 2141, 2186, 2262, 2319, 3163, 3223, 3272, 3300, 3425, 3519, 3625, 3672, 3798, 3826, 3883, 3929, 3984, 4035, 4101], '(156)': [272], 'Google': [273, 481, 505, 549, 645, 695, 858, 1464, 1512, 1591, 1665, 1729, 1769, 1909, 1949, 2006, 2052, 2143, 2188, 2264, 2321, 3165, 3225, 3274, 3302, 3427, 3521, 3627, 3674, 3800, 3828, 3885, 3931, 3986, 4037, 4103], 'Scholar': [274, 506, 550, 646, 696, 879, 1232, 1465, 1513, 1592, 1666, 1730, 1770, 1832, 2053, 2144, 2189, 2265, 2322, 2730, 2958, 3112, 3166, 3226, 3303, 3428, 3522, 3628, 3675, 3987, 4038, 4104], 'highly': [277], 'successful': [278], 'at': [279, 886, 908, 1392, 1620, 2034, 2303, 2608, 4191], 'ameliorating': [280], 'signs': [282], 'symptoms': [284, 3705], 'Treatment': [288], 'consisted': [289], 'weekly': [291], 'injections': [292, 307, 988, 2334], '165': [294], 'μg': [295], 'injected': [298, 895, 1142], 'directly': [299], 'into': [300, 896], 'vitreous': [302, 899], 'humor,': [303], 'maintenance': [306], 'every': [308, 916, 2324, 4130], 'other': [309, 2588], 'week': [310, 1019, 4131], 'until': [311], 'evidence': [312], 'progression.': [315], 'median': [317], 'time': [318, 2917, 3471], 'to': [319, 399, 437, 470, 494, 574, 611, 655, 662, 684, 708, 714, 727, 820, 942, 981, 1032, 1056, 1062, 1122, 1248, 1265, 1389, 1523, 1550, 1605, 1673, 1734, 1743, 1986, 2244, 2330, 2370, 2374, 2420, 2487, 2556, 2595, 2660, 2770, 2851, 2863, 2871, 2897, 2912, 2998, 3009, 3016, 3036, 3084, 3228, 3336, 3341, 3348, 3450, 3527, 3631, 3687, 3698, 3733, 4113, 4209, 4216, 4219, 4284], 'disease': [320, 737, 1330, 1343, 1391, 3124, 3133, 3700, 4262], 'progression': [321, 349], 'fomivirsen-treated': [324], 'group': [325, 338, 2392, 2878], '(n': [326, 2445, 4116], '=': [327, 340, 2401, 2446, 2463, 4117], '18)': [328], '71': [330], 'days': [331, 334], 'versus': [332, 356, 1140, 2393, 2502, 2507, 2630, 2647, 3505, 3915, 4021], '13': [333], 'untreated': [337, 360, 2070], '(p': [339, 989, 1144, 2467, 2632, 4203], '0.0001).': [341], 'Overall': [342], 'during': [343], 'course': [345], 'study,': [348, 2554], 'occurred': [350], '44%': [352], 'treated': [355, 1399, 2434, 2563, 2651, 4047], '70%': [357], 'patients.': [361, 1104, 2652, 4244, 4265], 'FDA': [367, 726, 1036, 1150, 2839, 2949, 3103, 4309], '1998': [369], 'EMEA': [372], '(European': [373], 'Agency': [374, 2696], 'Evaluation': [377], 'Medicinal': [379], 'Products)': [380], '1999.': [382], 'At': [383, 751, 2915], 'there': [386, 1107, 4268], 'high': [389, 1334, 1516, 2035, 2304, 2609], 'unmet': [390], 'need': [391], 'an': [393, 434, 698, 1431, 1548, 1597, 1876, 2021, 2110, 2290, 2456, 2557, 3011, 3410, 3565, 3760, 3868, 4308], 'anti-cytomegalovirus': [394], 'drug.': [396, 1069], 'Subsequently,': [397], 'due': [398, 2769, 2862], 'high-activity': [403], 'anti-retroviral': [404], 'therapy': [405, 1993, 2251], '(HAART),': [406], 'number': [408, 927], 'cases': [411, 1073, 1083], 'dramatically': [412, 1470], 'decreased.': [413], 'stopped': [415], '2002': [420], 'Europe': [422], '2006': [425], 'United': [428, 1314], 'States.': [429], 'Macugen': [430, 721, 1155, 1165, 1235, 1293], '(formerly': [431], 'pegaptanib)': [432], 'aptamer': [435, 535], 'targeted': [436, 533, 1604, 2637, 3035, 3630], 'vascular': [438, 538, 617, 628, 685, 769], 'endothelial': [439, 618, 686], 'growth': [440, 619, 687, 760], 'factor': [441, 620], '(VEGF165).': [442, 621], 'molecule': [444, 567], 'produced': [447], 'systematic': [450], 'evolution': [451, 462], 'ligands': [453, 464, 469, 683], 'exponential': [455, 466], 'enrichment': [456], '(SELEX)2Tuerk': [457], 'C.': [458, 673, 1956, 1960, 2214, 2218, 3585, 3944], 'Gold': [459, 511], 'L.': [460, 605, 3245, 3249, 3379, 3579, 3771, 3775, 3837, 3938], 'Systematic': [461], 'enrichment:': [467], 'RNA': [468, 682], 'bacteriophage': [471], 'T4': [472], 'DNA': [473], 'polymerase.Science.': [474], '1990;': [475], '249:': [476], '505-510Crossref': [477], '(7993)': [480], 'Scholar,': [482, 859, 1910, 1950, 2007, 3275, 3801, 3829, 3886, 3932], '3Eaton': [483], 'B.E.': [484], 'joys': [486], 'vitro': [489, 3748], 'selection:': [490], 'chemically': [491], 'dressing': [492], 'satiate': [495], 'protein': [496, 1538, 1686, 1772, 3235, 3320, 3758], 'targets.Curr.': [497], 'Opin.': [498], 'Chem.': [499, 638, 1658], 'Biol.': [500, 637], '1997;': [501], '1:': [502], '10-16Crossref': [503], 'strategy': [507], 'pioneered': [508], 'Larry': [510], '(an': [512], 'excellent': [513], 'review': [514], 'Ng': [516], 'et': [517, 1429, 1555, 1874, 2108, 3256, 3392, 3594, 3782, 3850, 3953], 'al.4Ng': [518], 'E.W.': [519], 'Shima': [520], 'D.T.': [521], 'Calias': [522], 'P.': [523, 1653, 3593, 3657, 3952], 'Cunningham': [524, 828], 'Jr.,': [525, 829], 'E.T.': [526, 830], 'Guyer': [527, 833], 'D.R.': [528, 834], 'Adamis': [529, 826], 'A.P.': [530, 827], 'Pegaptanib,': [531], 'anti-VEGF': [534, 1280], 'ocular': [537, 866, 898], 'disease.Nat.': [539], 'Rev.': [540, 1761], 'Discov.': [542, 3293, 3819, 4094], '2006;': [543], '5:': [544], '123-132Crossref': [545], '(1125)': [548], 'provides': [551], 'brief,': [553], 'concise': [554], 'description': [555], 'SELEX': [558], 'approach).': [559], 'Pegaptanib': [560, 660], '27': [562], 'nucleobases': [563], 'length.': [565], '3′-3′': [571], 'deoxythymidine': [572], 'cap': [573], 'promote': [575], 'nuclease': [576], 'stability;': [577], 'purine': [581], 'ribose': [582, 589], 'sugars': [583, 590], 'are': [584, 1520, 1800, 2974, 2987, 3033, 3313, 3474, 3544], '2′-O-methylated': [585], 'pyrimidine': [588], '2′-fluorinated.5Ruckman': [592], 'Green': [594, 670], 'L.S.': [595, 671], 'Beeson': [596], 'Waugh': [598], 'S.': [599, 1428, 1565, 1873, 1926, 2013, 2107, 2165, 2282, 3253, 3373, 3381, 3385, 3581, 3591, 3779, 3831, 3839, 3843, 3940, 3950], 'Gillette': [600], 'W.L.': [601], 'Henninger': [602], 'D.D.': [603], 'Claesson-Welsh': [604], 'Janjić': [606, 674], 'N.': [607, 675, 3480, 3647, 3890, 3996], '2′-Fluoropyrimidine': [608], 'RNA-based': [609], 'aptamers': [610], '165-amino': [613], 'acid': [614], 'form': [615], 'Inhibition': [622, 676, 836], 'receptor': [624, 678, 1746], 'binding': [625, 665, 679, 707, 1742, 3332], 'VEGF-induced': [627], 'permeability': [629], 'through': [630, 1240, 1740], 'interactions': [631], 'requiring': [632], 'exon': [634, 3063, 3086, 3566, 3636, 3641, 3663], '7-encoded': [635], 'domain.J.': [636], '1998;': [639], '273:': [640], '20556-20567Crossref': [641], '(647)': [644], 'A': [647, 2492], '40': [648], 'kDa': [649], 'polyethylene': [650], 'glycol': [651], 'substituent': [652], 'linked': [654], 'molecular': [658, 3554], 'terminus.': [659], 'binds': [661], 'heparin': [664], 'site': [666, 2776], 'VEGF1656Jellinek': [668], 'D.': [669, 1424, 1869, 1958, 2103, 2216, 3583, 3942], 'Bell': [672], 'high-affinity': [681], 'factor.Biochemistry.': [688], '1994;': [689], '33:': [690], '10450-10456Crossref': [691], '(162)': [694], 'affinity': [699], 'picomolar': [702], 'range': [703], 'inhibits': [705], 'both': [709, 1352, 2087, 3730], 'VEGFR1': [710], 'VEGFR2,': [712], 'though': [713, 1299, 2481, 2580, 3680], 'lesser': [716, 2422, 2490, 2597], 'extent': [717, 2423], 'latter.': [720], 'treat': [728], 'age-related': [729, 846], 'macular': [730, 847], 'degeneration': [731], '(AMD)': [732], 'retina.': [735], 'leading': [740, 3227], 'cause': [741, 3195], 'blindness': [743], 'people': [745], 'over': [746, 4152, 4303], 'age': [748, 1406, 2081, 3718], '50.': [750], 'least': [752, 887], 'part,': [754], "it's": [755], 'caused': [756], 'VEGF165-stimulated': [759], 'blood': [762], 'vessels': [763], '(neovascularization)': [764], 'choroid': [767], '(the': [768, 3326], 'tissue': [770, 3370], 'beneath': [771, 3323], 'retina)': [773], 'eye.': [776], 'Disease': [777], 'activity': [778, 1633], 'especially': [780], 'severe': [781, 1935, 2032, 2174, 2301, 2606, 2746, 3710], 'macula': [784], 'retina': [787], 'can': [789, 1468, 3006, 3706, 3723], 'result': [790, 3462], 'central': [792, 1733], 'blindness.': [793], 'safety': [795, 1990, 2248], 'efficacy': [797, 871, 1988, 2246, 2679, 4072], 'pegaptanib': [799, 1028, 1049, 1118], 'two': [803], 'identical': [804], 'These': [806, 1070, 2985, 3184], 'were': [808, 881, 962, 1050, 1054, 1099, 1120, 1236, 1618, 1853, 2327, 2344, 2354, 2425, 2433, 2479, 2485, 2573, 2583, 2593, 2658, 2672, 2908, 2966, 4046, 4106, 4110, 4167, 4182, 4269], 'prospective,': [809], 'randomized,': [810, 1982, 2039, 2240, 2308], 'double-masked,': [811], 'multi-centered,': [812], 'dose': [814, 1041, 1517, 4288], 'finding': [815], 'referred': [819], 'VISION': [823], 'trials.7Gragoudas': [824], 'E.S.': [825], 'Feinsod': [831], 'M.': [832, 1968, 2019, 2226, 2288, 3377, 3575, 3587, 3835, 3934, 3946], 'VEGF': [835, 862], 'Ocular': [839], 'Neovascularization': [840], 'Clinical': [841], 'Trial': [842, 2364], 'GroupPegaptanib': [843], 'neovascular': [845], 'degeneration.N.': [848], 'Engl.': [849], 'Med.': [851], '2004;': [852], '351:': [853], '2805-2816Crossref': [854], '(2103)': [857], "8D'Amico": [860], 'D.J.': [861, 1414, 1859, 2093, 3391, 3651, 3849], 'inhibition': [863, 1972, 2230], 'neovascularization': [867], '(VISION):': [868], 'Second': [869], 'year': [870], 'data.Invest.': [872], 'Vis.': [874], 'Sci.': [875], '2005;': [876, 1585], '46:': [877, 1586], '2309Google': [878], 'There': [880, 961], '1,186': [882], 'received': [885, 930, 2532, 2536, 4137, 4144, 4160], 'one': [888, 3038], 'pegatinib.': [891], 'humor': [900], '(after': [901], 'application': [903], 'appropriate': [906], 'anesthesia)': [907], 'doses': [909, 976, 1026, 1047], '0.3,': [911, 971], '1.0,': [912, 972], 'or': [913, 1007, 2069, 2082, 2340, 3015, 3047, 4126, 4148, 4287], '3.0': [914, 974], 'mg': [915, 975, 1040, 2338], '6': [917], 'weeks': [918, 921, 2406], '48': [920], '(nine': [922], 'treatments': [923], 'total).': [924], 'An': [925, 1568], 'equivalent': [926], 'sham': [932, 987, 1141], 'treatment.': [933, 2411, 2624], 'Efficacy': [934], 'determined': [936], 'ability': [939, 3732], 'visually': [943], 'read': [944], 'letters': [946, 1003], 'on': [947, 1011, 1496, 1713, 1747, 2346, 2833, 3508, 3918, 4024, 4187], 'eye': [948, 1014], 'charts,': [949], 'metric': [951, 4065], 'currently': [954, 1219, 4067], 'standard': [956], 'ophthalmology': [959], '70%,': [963], '71%,': [964], '65%': [966], 'receiving': [969, 985, 1937, 2176], 'pegaptanib,': [978], 'respectively,': [979], 'compared': [980, 2454, 2555, 4237], '55%': [982], '>': [990, 992, 995], '0.001,': [991, 993], '0.03,': [996], 'respectively)': [997], 'lost': [999, 4186, 4235], 'less': [1000, 1287, 3709, 4197], 'than': [1001, 1067, 1269, 2812, 2970, 3711, 4198], 'visual': [1005], 'acuity,': [1006], 'three': [1009, 4114], 'lines': [1010], 'chart,': [1015], '54': [1018], 'duration': [1020], 'trial.': [1023], 'Because': [1024, 1367, 4155], 'higher': [1025], 'did': [1029, 2826], 'not': [1030, 1317, 1398, 1402, 2687, 2827, 2929, 2975, 3525], 'lead': [1031], 'greater': [1033, 2811], 'activity,': [1034], '0.3': [1039], 'late': [1043], '2004.': [1044], 'All': [1045, 1095, 2342], 'safe.': [1051], 'Side': [1052], 'effects': [1053], 'mild': [1055], 'moderate': [1057], 'intensity': [1059, 1518], 'related': [1061], 'injection': [1064, 2775], 'process': [1065], 'rather': [1066], 'included': [1071], 'five': [1072], 'each': [1074], 'traumatic': [1076], 'injury': [1077], 'lens': [1079], 'detachment': [1080], '12': [1082, 2464], 'endophthalmitis': [1085], '(most': [1086], 'nevertheless': [1090], 'remained': [1091], 'trial).': [1094], 'adverse': [1097, 2771, 4275, 4281], 'events': [1098, 2772, 2804, 4282], 'seen': [1100, 2498], 'pegaptanib-injected': [1103], 'As': [1105, 2585, 3743], 'anticipated,': [1106], 'no': [1109, 4270], 'systemic': [1110, 3405, 3863], 'toxicity.': [1111, 2780], 'results': [1113, 1356, 1979, 2237, 3360, 4178], 'after': [1114, 1125, 2407, 2805, 3404, 3862], '2': [1115, 2766], 'similar': [1121, 2659, 3686], 'those': [1123, 2562, 3542, 3712], 'observed': [1124, 2662, 2755, 4280], '1': [1126], 'year,': [1127], '45%': [1130], 'mean': [1131, 2377, 2543], 'relative': [1132], 'change': [1134, 2379], 'vision': [1136], 'noted': [1137], '<': [1145, 2468, 2578, 2633, 4204], '0.01).': [1146], 'However,': [1147, 1191, 1242, 2751, 2832, 4290], 'since': [1148], 'approval': [1151, 2841], 'date': [1152], '2004,': [1154], 'has': [1156, 3114], 'led': [1157, 4283], 'peripatetic': [1159], 'existence.': [1160], 'Initially': [1161], 'NeXstar,': [1164], 'eventually': [1167, 4159], 'acquired': [1168, 1187, 1210, 1227], 'Eyetech': [1170, 1184, 1195], 'Pharmaceuticals,': [1171], 'Pfizer': [1176], 'above.': [1181], '2005,': [1183], 'Pharmaceuticals': [1185, 1213], 'OSI': [1189, 1201], 'Pharmaceuticals.': [1190], '2008,': [1193], 'former': [1194], 'employees': [1196], 'purchased': [1197], 'established': [1203], 'Eyetech,': [1204], 'Inc.,': [1205], 'turn': [1208], 'Valeant': [1212], '2010.': [1215], 'marketed': [1220], 'Bausch': [1222], 'Lomb,': [1224], '2013': [1229, 2836], 'Valeant.9http://ophthalmologytimes.modernmedicine.com/ophthalmologytimes/news/modernmedicine/modern-medicine-news/valeant-acquires-eyetech.Google': [1231], 'Sales': [1233], 'satisfactory': [1237], '2004': [1239], '2011.': [1241], '2010,': [1244], 'sales': [1245], 'had': [1246, 2361, 2901, 3753, 4319], 'declined': [1247], 'only': [1249, 4229], '$12': [1250], 'million/year': [1251], 'face': [1254], 'stiff': [1256], 'competition': [1257, 2864], 'Lucentis': [1259], '(ranibizumab;': [1260], 'Novartis).': [1261], 'appears': [1264], 'be': [1266, 1469, 3007, 3707, 3724], 'more': [1267, 2969, 3569], 'effective': [1268], 'Macugen.': [1270], 'Competition': [1271], 'also': [1272, 1773, 2426, 2574, 2754, 2784, 4143], 'came': [1273], 'off-label': [1276], 'use': [1277], 'mAb': [1281], 'bevacizumab': [1282], '(Avastin;': [1283], 'Genentech),': [1284], 'expensive.': [1288], 'Nevertheless,': [1289, 2847], 'still': [1294], 'retains': [1295], 'relatively': [1297], 'small,': [1298], 'somewhat': [1300, 2673], 'precarious,': [1301], 'market': [1302, 2898], 'share': [1303], 'AMD.': [1308], 'Mipomersen': [1309, 2054, 2625], 'States': [1315], '(but': [1316], 'European': [1320, 2694], 'Union)': [1321], 'homozygous': [1323, 1445, 1890, 2124], 'familial': [1324, 1446, 1483, 1700, 1891, 1977, 2125, 2235], 'hypercholesterolemia': [1325, 1484, 1701, 1936, 2033, 2175, 2302, 2607, 2748], '(HoFH).': [1326], 'associated': [1331, 1791], 'very': [1333, 1394, 1781], 'plasma': [1335, 1363], 'concentration': [1336], 'low-density': [1338, 1782], 'lipoprotein': [1339, 1689, 1756, 1783], '(LDL).': [1340], 'Molecularly,': [1341], 'distinguished': [1345], 'loss': [1347, 3233, 3357], 'function': [1349, 3440], 'mutations': [1350, 3171, 3185, 3312], 'LDL-receptor': [1353], 'genes.': [1354], 'reduced': [1359, 1471, 2450, 2486, 2575, 2594, 2626, 2737], 'liver': [1360, 1788, 2779], 'uptake': [1361], 'LDL': [1364, 1439, 1541, 1574, 1745, 1804, 1884, 2118], 'cholesterol': [1365, 1440, 1575, 1885, 2119, 2671, 2824], '(LDL-C).': [1366], 'lack': [1370], 'clearance': [1372, 1736], 'LDL-C,': [1374, 1553, 2664], 'ailment': [1378, 3549], 'develop': [1379], 'cutaneous': [1380], 'tendinous': [1382], 'xanthomatas': [1383], '(benign': [1384], 'cholesterol-laden': [1385], 'tumors),': [1386], 'addition': [1388], 'cardiovascular': [1390, 2036, 2305, 2610, 2803, 2830], 'young': [1395, 3118, 3431], 'age.': [1396, 3443], 'Patients': [1397, 4135], 'usually': [1400], 'do': [1401], 'live': [1403, 3139], 'beyond': [1404], '30.10Raal': [1409], 'F.J.': [1410, 1855, 2089], 'Santos': [1411, 1856, 2090], 'R.D.': [1412, 1857, 2091], 'Blom': [1413, 1858, 2092], 'Marais': [1415, 1860, 2094], 'A.D.': [1416, 1861, 2095], 'Charng': [1417, 1862, 2096], 'M.J.': [1418, 1559, 1863, 2097], 'Cromwell': [1419, 1864, 2010, 2098, 2279], 'W.C.': [1420, 1865, 2011, 2099, 2280], 'Lachmann': [1421, 1866, 2100], 'R.H.': [1422, 1867, 2101], 'Gaudet': [1423, 1868, 1957, 2102, 2215], 'Tan': [1425, 1870, 2104], 'J.L.': [1426, 1871, 2105], 'Chasan-Taber': [1427, 1872, 1925, 2106, 2164], 'al.Mipomersen,': [1430, 1875, 2109], 'apolipoprotein': [1432, 1544, 1569, 1877, 2022, 2111, 2291], 'B': [1433, 1545, 1570, 1878, 1970, 2023, 2112, 2228, 2292], 'synthesis': [1434, 1879, 1971, 2024, 2113, 2229, 2293], 'inhibitor,': [1435, 1880, 2025, 2114, 2294], 'lowering': [1437, 1882, 2116, 2350], 'concentrations': [1441, 1886, 2120], 'hypercholesterolaemia:': [1447, 1892, 2126], 'randomised,': [1449, 1894, 2128], 'double-blind,': [1450, 1895, 1983, 2040, 2129, 2241, 2309], 'placebo-controlled': [1451, 1896, 1928, 1984, 2041, 2130, 2167, 2242, 2310], 'trial.Lancet.': [1452, 1897, 2131], '2010;': [1453, 1898, 2132], '375:': [1454, 1899, 2133], '998-1006Abstract': [1455, 1900, 2134], '(745)': [1463, 1908, 2142], 'LDL-C': [1466, 2071, 2148, 2195, 2270, 2384, 2448, 2496, 2538, 2627, 2638, 2739], 'levels': [1467, 2385, 2484, 2572, 2582, 2592, 2825], 'statins,11Besseling': [1473], 'Sjouke': [1475, 1692], 'B.': [1476, 1693], 'Kastelein': [1477, 1694], 'J.J.P.': [1478, 1695], 'Screening': [1479, 1696], '-': [1485, 1702], 'Lessons': [1486, 1703], 'opportunities': [1491, 1708], 'future': [1494, 1711], '(based': [1495, 1712], 'Anitschkow': [1498, 1715], 'Lecture': [1499, 1716], '2014).Atherosclerosis.': [1500, 1717], '2015;': [1501, 1718], '241:': [1502, 1719], '597-606Abstract': [1503, 1720], '(32)': [1511, 1728], 'but': [1514, 2665], 'even': [1515], 'statins': [1519], 'often': [1521, 3445], 'insufficient': [1522], 'reach': [1524], 'therapeutic': [1525, 3029], 'goals;': [1526], 'i.e.,': [1527], 'elimination': [1528], 'risk': [1531], 'coronary': [1533, 1997, 2207, 2255], 'heart': [1534], 'disease.': [1535], 'core': [1537], 'particle': [1542], '(apoB).': [1546], 'attempt': [1549], 'diminish': [1551], 'circulating': [1552], 'Crooke': [1554], 'al.,12Crooke': [1556], 'R.M.': [1557], 'Graham': [1558], 'Lemonidis': [1560], 'K.M.': [1561], 'Whipple': [1562], 'C.P.': [1563], 'Koo': [1564], 'Perera': [1566], 'R.J.': [1567], 'lowers': [1573], 'hyperlipidemic': [1577], 'mice': [1578], 'without': [1579], 'causing': [1580], 'hepatic': [1581, 2792], 'steatosis.J.': [1582], 'Lipid': [1583, 1811, 2709], 'Res.': [1584, 1659], '872-884Crossref': [1587], '(206)': [1590], 'synthesized': [1593], 'mipomersen': [1594, 1931, 1974, 2170, 2232, 2339, 2371, 2391, 2436, 2506, 2515, 2533, 2549, 2613, 2685, 2806, 2820, 2927], '(Isis': [1595], '147764),': [1596], '20-mer': [1599], '2′-methoxyethoxy': [1601], '(MOE)': [1602], 'gapmer': [1603], 'coding': [1607], 'region': [1608, 3634], 'apoB': [1611, 1640, 1682, 2655], '(see': [1613, 2477], 'below).': [1614], 'MOE': [1616, 1646], 'groups': [1617], 'incorporated': [1619], 'positions': [1621], '1–5': [1622], '15–20': [1624], 'chain,': [1628], 'where': [1629], 'they': [1630, 3442], 'block': [1631], 'exonuclease': [1632], 'increase': [1635, 2457, 2558], 'Tm': [1637], 'mRNA/ASO': [1641], 'duplex': [1642, 1670], '1.1°C': [1644], 'per': [1645], 'residue.13De': [1647], 'Mesmaeker': [1648], 'A.': [1649, 3643, 3645], 'Haener': [1650], 'R.': [1651, 1806, 1916, 1954, 2155, 2212, 2704], 'Martin': [1652], 'Moser': [1654], 'H.E.': [1655], 'Antisense': [1656, 3284, 3810, 4085], 'oligonucleotides.Acc.': [1657], '1995;': [1660], '28:': [1661], '366-374Crossref': [1662], '(480)': [1664], 'stabilizes': [1668], 'helps': [1672], 'enable': [1674], 'cleavage': [1675], 'RNase': [1680, 2982], 'H1.': [1681], 'critical': [1685], 'atherogenic': [1688, 2027, 2296], 'particles11Besseling': [1690], 'particles': [1739, 1785], 'hepatocytes.14Davidson': [1748], 'N.O.': [1749], 'Shelness': [1750], 'G.S.': [1751, 2009, 2278], 'APOLIPOPROTEIN': [1752], 'B:': [1753], 'editing,': [1755], 'assembly,': [1757], 'presecretory': [1759], 'degradation.Annu.': [1760], 'Nutr.': [1762], '2000;': [1763], '20:': [1764], '169-193Crossref': [1765], '(234)': [1768], 'plays': [1774], 'role': [1776], 'production': [1779], '(VLDL)': [1784], 'VLDL': [1794, 1796], 'particle.': [1795], 'particles,': [1797], 'turn,': [1799], 'metabolic': [1801, 2362], 'precursors': [1802], 'particles.15Mahley,': [1805], 'Biochemistry': [1807, 2705], 'Physiology': [1809, 2707], 'Lipoprotein': [1813, 2711], 'Metabolism.': [1814, 2712], 'Principles': [1816, 2714], 'Practice': [1818, 2716], 'Endocrinology': [1820, 2718], 'Metabolism,': [1822, 2720], 'Third': [1823, 2721], 'Edition,': [1824, 2722], 'K.L.': [1825, 2723], 'Becker,': [1826, 2724], 'ed.': [1827, 2725], '(Lippincott': [1828, 2726], 'Williams': [1829, 2727], 'Wilkins).Google': [1831, 2729], 'After': [1833, 4226], 'completion': [1834], 'studies': [1836, 3752], 'healthy': [1838], 'volunteers': [1839], 'phase': [1841, 1849, 3412, 3870, 3991], 'I': [1842], 'II': [1844], 'studies,': [1846], 'four': [1847], 'different': [1848], 'III': [1850, 3992], 'performed.10Raal': [1854], '16McGowan': [1911], 'M.P.': [1912, 2151], 'Tardif': [1913, 2152], 'J.C.': [1914, 2153, 3278, 3804, 4079], 'Ceska': [1915, 2154], 'Burgess': [1917, 2156], 'L.J.': [1918, 2157], 'Soran': [1919, 2158], 'H.': [1920, 2159], 'Gouni-Berthold': [1921, 2160], 'I.': [1922, 2161], 'Wagener': [1923, 2162], 'G.': [1924, 2163], 'Randomized,': [1927, 2166], 'maximally': [1938, 2177, 2347], 'tolerated': [1939, 2178, 2348], 'lipid-lowering': [1940, 2179], 'therapy.PLoS': [1941, 2180], 'ONE.': [1942, 2181], '2012;': [1943, 2000, 2182, 2258], '7:': [1944, 2183], 'e49006Crossref': [1945, 2184], '(184)': [1948, 2187], '17Stein': [1951], 'E.A.': [1952, 2210], 'Dufour': [1953, 2211], 'Gagne': [1955, 2213], 'East': [1959, 2217], 'Donovan': [1961, 2219], 'J.M.': [1962, 2220], 'Chin': [1963, 2014, 2221, 2283], 'W.': [1964, 2015, 2222, 2284], 'Tribble': [1965, 2223], 'D.L.': [1966, 2224], 'McGowan': [1967, 2225], 'Apolipoprotein': [1969, 2227], 'heterozygous': [1976, 2083, 2234, 2747], 'hypercholesterolemia:': [1978, 2236], 'assess': [1987, 2245], 'add-on': [1992, 2250], 'artery': [1998, 2208, 2256], 'disease.Circulation.': [1999, 2257], '126:': [2001, 2259], '2283-2292Crossref': [2002, 2260], '(249)': [2005, 2263], '18Thomas': [2008], 'Ali': [2012, 2281], 'Flaim': [2016, 2285], 'J.D.': [2017, 2286], 'Davidson': [2018, 2287], 'Mipomersen,': [2020, 2289], 'reduces': [2026, 2295], 'lipoproteins': [2028, 2297], 'risk:': [2037, 2306], 'trial.J.': [2042, 2311], 'Am.': [2043, 2312], 'Coll.': [2044, 2313], 'Cardiol.': [2045, 2314], '2013;': [2046, 2315, 3268, 3297, 3794, 3823, 4098], '62:': [2047, 2316], '2178-2184Crossref': [2048, 2317], '(181)': [2051, 2320], 'distinct': [2058], 'sets': [2059], 'patients:': [2061], '#1': [2063, 2365], 'genetic': [2065], 'confirmation': [2066], 'HoFH': [2068, 2743], '>500': [2072], 'mg/dL': [2073, 2197, 2202, 2272, 2642], 'either': [2075, 2336], 'xanthoma': [2076], 'before': [2077], '10': [2078], 'FH': [2084], '(HeFH)': [2085], 'parents;10Raal': [2088], '#2': [2146], '>200': [2149], 'mg/dL;16McGowan': [2150], '#3': [2191, 2525], 'HeFH': [2193], 'plus': [2194], '>100': [2196, 2271], '+': [2198, 2273], 'serum': [2199, 2274, 2383, 2472, 2482], 'triglycerides': [2200, 2275, 2668], '<200': [2201, 2276], 'diagnosis': [2205], 'disease;17Stein': [2209], '#4': [2268], 'mg/dL.18Thomas': [2277], 'trial,': [2325], '2:1': [2328], '26': [2331], 'weekly,': [2332], 'subcutaneous': [2333], '200': [2337], 'placebo.': [2341, 2375, 2440, 2537, 2565, 2813], 'maintained': [2345], 'lipid': [2349], 'therapy.': [2351], 'Most': [2352], 'middle': [2355], 'aged,': [2356], 'overweight,': [2357], '50%': [2360], 'syndrome.': [2363], '(HoFH)': [2366], 'assigned': [2367, 4112], '34': [2368], '17': [2373], 'percentage': [2378], 'baseline': [2381], '25%': [2388, 2512], '3%': [2395], 'placebo': [2398, 2465, 2617, 2650, 4141, 4157], 'group,': [2399], 'p': [2400, 2577], '0.0003,': [2402], 'measured': [2404], '28': [2405], 'initiation': [2409], 'Similar': [2412], 'changes': [2413], 'total': [2415, 2670], 'cholesterol,': [2416, 2418, 2567], 'non-HDL': [2417], 'and,': [2419], 'triglycerides,': [2424], 'seen.': [2427], '#2,': [2430], '39': [2431], '19': [2438], 'mipomersen-treated': [2443], '27),': [2447], '36%,': [2452], '13%': [2460, 3558], 'n': [2462], '0.001).': [2469], 'Changes': [2470], 'apoB,': [2473, 2568], 'non-HDL-C,': [2474, 2569], 'Lp(a)': [2476, 2571], 'below)': [2478], 'similar,': [2480], 'triglyceride': [2483, 2591], 'much': [2489, 2853, 3708], 'extent.': [2491, 2598], '>15%': [2493], 'decrease': [2494], 'almost': [2500], '80%': [2501], '17%': [2503], 'placebo-treated': [2508], 'achieved': [2517, 2644], '>50%': [2519], 'reduction': [2520, 2822], 'LDL-C.': [2522], '(HeFH),': [2526], '83': [2527], '41': [2535], 'decreased': [2540], '28%': [2545], '73': [2548], 'who': [2551, 3433, 3543], 'completed': [2552, 2623], '5%': [2560], 'Total': [2566], '(all': [2576], '0.001),': [2579], 'HDL-C': [2581], 'unaltered.': [2584], 'above,': [2590], 'Finally,': [2599], '#4,': [2602], 'risk,': [2611], '60': [2612], '44': [2616], '(out': [2618], '158': [2620], 'randomized)': [2621], '36%': [2629], '4.5%': [2631], '0.001)': [2634], 'level': [2639], '<100': [2641], '76%': [2646], '38%': [2648], 'Reductions': [2653], 'non-HDL-C': [2657], 'reductions': [2666], 'smaller.': [2674], 'Despite': [2675], 'wealth': [2677], 'data,': [2680], 'initial': [2681], 'opinion': [2683, 4252], 'favorable.': [2688], 'On': [2689], 'December': [2690], '13,': [2691, 2835], '2012,': [2692], 'Medicines': [2695], '(EMA)': [2697], 'refused': [2698], 'authorization': [2700], 'drug.15Mahley,': [2703], 'EMA': [2732, 2782, 2815], 'acknowledged': [2733], 'average': [2738], '25%–36%.': [2750], 'agency': [2753], '"high': [2758], 'proportion"': [2759], 'discontinued': [2762], 'mostly': [2768], 'such': [2773], 'reactions': [2777], 'concerned': [2785], 'increasing': [2788], 'possibility': [2789], 'developing': [2791, 2801], 'steatosis': [2793], '(fatty': [2794], 'liver),': [2795], 'probability': [2799], 'serious': [2802, 4274], 'fact': [2810], 'therefore': [2816], 'concluded': [2817], 'induced': [2821], 'outweigh': [2828], 'risk.': [2831], 'U.S.': [2838], 'granted': [2840], 'Kynamro.': [2846], 'failed': [2850], 'generate': [2852], 'way': [2856], 'sales.': [2858], 'probably': [2861], 'small': [2867], 'molecule,': [2868], 'seems': [2870], 'garnered': [2873], 'most': [2874], 'tiny': [2877], 'HoFH.': [2882], 'By': [2883, 2903], '2016,': [2887, 2905, 3989], '(now': [2892], 'renamed': [2893], 'Ionis)': [2894], 'Genzyme': [2896], 'dissolved.': [2902], 'May,': [2904], "Kynamro's": [2906], 'rights': [2907], 'sold': [2909], 'Ionis': [2911], 'Kastle': [2913], 'Therapeutics.': [2914], 'writing': [2920], '(January,': [2921], '2017),': [2922], 'fate': [2925], 'clear.': [2930], 'Eteplirsen': [2931, 2942, 3081, 3096, 3113, 3493, 3903, 4009], '(Sarepta)': [2932], '30-mer': [2935], 'phosphomorpholidate': [2936], 'oligonucleotide.19Excerpted': [2937], 'from:': [2938, 3092], 'Stein,': [2939, 3093], 'C.A.': [2940, 3094, 3144, 3204], '(2016).': [2941, 3095], 'Duchenne': [2945, 3099, 3127, 3155, 3215, 3264, 3290, 3401, 3511, 3606, 3790, 3816, 3859, 3921, 3965, 4027, 4091], 'muscular': [2946, 3100, 3128, 3156, 3216, 3265, 3291, 3402, 3512, 3607, 3693, 3791, 3817, 3860, 3922, 3966, 4028, 4092], 'dystrophy:': [2947, 3101, 3608, 3967], 'faces': [2950, 3104], 'difficult': [2952, 3106], 'choice.': [2953, 3107], 'Mol.': [2954, 3108], 'Ther.': [2955, 3109, 3295, 3667, 3821, 4096], '24,': [2956, 3110], '1884–1885.Google': [2957, 3111], 'Unlike': [2959], 'charged': [2960], 'oligonucleotides,': [2962], 'uncharged': [2963], 'phosphomorpholidates,': [2964], 'introduced': [2968], '35': [2971], 'ago,': [2973], 'substrates': [2976], 'nucleases': [2978], 'cannot': [2980], 'elicit': [2981], 'H': [2983], 'activity.': [2984, 2992], 'properties': [2986], 'important': [2988], 'oligonucleotide-induced': [2990], 'splice-switching': [2991], 'Oligonucleotide-directed': [2993], 'splice': [2994, 3050], 'switching': [2995], 'used': [2997, 4168], 'manipulate': [2999], 'alternative': [3000], 'pre-mRNA': [3001], 'splicing.': [3002], 'Splice-switching': [3003], 'oligos': [3004], '(SSOs)': [3005], 'employed': [3008], 'correct': [3010], 'irregular': [3012], 'splicing': [3013, 3023, 3042, 3068], 'event': [3014], 'induce': [3017], 'expression': [3019, 3150, 3210, 3599, 3958], 'new': [3022], 'variant,': [3024], 'may': [3026], 'function.': [3030], 'SSOs': [3032], 'generally': [3034], 'bind': [3037], 'key': [3041], 'sequences': [3043], '(usually': [3044], 'donor': [3046], 'acceptor': [3049], 'site)': [3051], 'define': [3053], "exons'": [3055], 'boundaries': [3056], 'within': [3057], 'gene': [3059], 'pre-mRNA.': [3060], 'When': [3061], 'boundary': [3064], 'masked,': [3066], 'machinery': [3069], 'searches': [3070], 'downstream': [3071], 'another': [3073], 'suitable': [3074], 'site,': [3075], 'thus': [3076], 'skipping': [3077, 3394, 3639, 3664, 3852], 'problematic': [3079], 'exon.': [3080], 'designed': [3083], 'skip': [3085], '51': [3087], 'dystrophin': [3090, 3149, 3182, 3192, 3209, 3319, 3359, 3396, 3598, 3684, 3757, 3854, 3957], 'protein.19Excerpted': [3091], 'male': [3119, 3140], 'devastating': [3123], 'known': [3125], 'dystrophy': [3129, 3403, 3861], '(DMD).': [3130], 'X-linked': [3132], 'found': [3135, 3690], '1/3,500–1/5,000': [3138], 'births20Robinson-Hamm': [3141], 'J.N.': [3142, 3202], 'Gersbach': [3143, 3203], 'Gene': [3145, 3205, 3666], 'therapies': [3146, 3206], 'restore': [3148, 3208], 'dystrophy.Hum.': [3157, 3217], 'Genet.': [3158, 3218], '2016;': [3159, 3219, 3515, 3925, 4031], '135:': [3160, 3220], '1029-1040Crossref': [3161, 3221], '(50)': [3164, 3224], 'characterized': [3169], '(such': [3172], 'intragenic': [3174], 'deletions,': [3175], 'duplications,': [3176], 'point': [3178], 'mutations)': [3179], 'gene.': [3183], 'disrupt': [3186], 'reading': [3188], 'frame': [3189], 'introduction': [3197], 'premature': [3199], 'stop': [3200], 'codons,20Robinson-Hamm': [3201], 'degradation': [3230], 'synthesis21Mendell': [3236], 'J.R.': [3237, 3478, 3763, 3888, 3994], 'Rodino-Klapac': [3238, 3764], 'L.R.': [3239, 3765], 'Sahenk': [3240, 3766], 'Z.': [3241, 3767], 'Roush': [3242, 3768], 'K.': [3243, 3383, 3486, 3653, 3769, 3841, 3896, 4002], 'Bird': [3244, 3770], 'Lowes': [3246, 3481, 3772, 3891, 3997], 'L.P.': [3247, 3482, 3773, 3892, 3998], 'Alfano': [3248, 3483, 3774, 3893, 3999], 'Gomez': [3250, 3776], 'A.M.': [3251, 3649, 3777], 'Lewis': [3252, 3778], 'Kota': [3254, 3780], 'al.Eteplirsen': [3257, 3783], 'GroupEteplirsen': [3259, 3785], 'dystrophy.Ann.': [3266, 3513, 3792, 3923, 4029], 'Neurol.': [3267, 3514, 3615, 3793, 3924, 3974, 4030], '74:': [3269, 3795], '637-647Crossref': [3270, 3796], '(538)': [3273, 3799], '22van': [3276, 3802], 'Deutekom': [3277, 3803, 4078], 'de': [3279, 3805, 4080], 'Kimpe': [3280, 3806, 4081], 'S.J.': [3281, 3807, 4082], 'Campion': [3282, 3808, 4083], 'G.V.': [3283, 3809, 4084], 'personalized': [3287, 3813, 4088], 'medicine': [3288, 3814, 4089], 'dystrophy.Drug': [3292, 3818, 4093], 'Today': [3294, 3820, 4095], 'Strateg.': [3296, 3822, 4097], '10:': [3298, 3824, 4099], 'e149-e156Crossref': [3299, 3825, 4100], '(7)': [3301, 3827, 4102], 'striated': [3305], 'muscle.': [3306], 'consequences': [3309], 'catastrophic': [3314], 'patient.': [3317], 'located': [3322], 'sarcolemma': [3325], 'muscle': [3327, 3350, 3363, 3456], 'cell': [3328, 3351], 'membrane).': [3329], 'Via': [3330], 'N-terminal': [3335], 'F-actin': [3337], 'C-terminal': [3340], 'β-dystroglycan,': [3342], 'it': [3343], 'connects': [3344], 'cellular': [3346], 'cytoskeleton': [3347], 'membrane,': [3352], 'sarcolemma.': [3354], 'Histologically,': [3355], 'inflammation,': [3362], 'degeneration,': [3364], 'replacement': [3367], 'fibrotic': [3369], 'fat.23Cirak': [3372], 'Arechavala-Gomeza': [3374, 3576, 3832, 3935], 'V.': [3375, 3577, 3833, 3936], 'Guglieri': [3376, 3586, 3834, 3945], 'Feng': [3378, 3578, 3836, 3937], 'Torelli': [3380, 3838], 'Anthony': [3382, 3840], 'Abbs': [3384, 3590, 3842, 3949], 'Garralda': [3386, 3844], 'M.E.': [3387, 3845], 'Bourke': [3388, 3846], 'Wells': [3390, 3650, 3848], 'al.Exon': [3393, 3851], 'restoration': [3397, 3596, 3755, 3855, 3955], 'phosphorodiamidate': [3406, 3864], 'morpholino': [3407, 3602, 3865, 3961], 'oligomer': [3408, 3603, 3866, 3962], 'treatment:': [3409, 3867], 'open-label,': [3411, 3869], '2,': [3413, 3871], 'dose-escalation': [3414, 3872], 'study.Lancet.': [3415, 3873], '2011;': [3416, 3874], '378:': [3417, 3875], '595-605Abstract': [3418, 3876], '(697)': [3426, 3884], 'Children': [3429], 'adults': [3432], 'suffer': [3434], 'DMD': [3436, 3472, 3499, 3537, 3909, 4015, 4045], 'progressively': [3437], 'lose': [3438], 'neuromuscular': [3439], 'culminates': [3446], 'their': [3448, 3453, 3476, 3731, 3736, 4251], 'inability': [3449], 'walk': [3451, 4061], 'mid-teens.': [3454], 'Sadly,': [3455], 'deterioration': [3457], 'progressive:': [3459], 'inevitable': [3461], 'cardiomyopathy,': [3464], 'respiratory': [3465], 'failure,': [3466], 'death': [3468], 'twenties.24Mendell': [3477], 'Goemans': [3479, 3646, 3889, 3995], 'L.N.': [3484, 3894, 4000], 'Berry': [3485, 3895, 4001], 'Shao': [3487, 3897, 4003], 'Kaye': [3489, 3899, 4005], 'E.M.': [3490, 3900, 4006], 'Mercuri': [3491, 3901, 4007], 'E.': [3492, 3589, 3655, 3902, 3948, 4008], 'Group': [3495, 3905, 4011], 'Telethon': [3497, 3907, 4013], 'Foundation': [3498, 3908, 4014], 'Italian': [3500, 3910, 4016], 'NetworkLongitudinal': [3501, 3911, 4017], 'effect': [3502, 3912, 4018], 'eteplirsen': [3504, 3914, 4020, 4123, 4254], 'historical': [3506, 3916, 4022, 4165, 4201, 4224, 4242], 'control': [3507, 3917, 4023, 4243], 'ambulation': [3509, 3919, 4025], '79:': [3516, 3926, 4032], '257-271Crossref': [3517, 3927, 4033], '(363)': [3520, 3930, 4036], 'It': [3523], 'possible': [3526], 'express': [3528], 'words': [3530], 'anguish': [3532], 'parents': [3535], 'nor': [3539], 'suffering': [3541], 'afflicted': [3545], 'terrible': [3548], 'must': [3550], 'stoically': [3551], 'endure.': [3552], 'lesion,': [3555], 'DMD,': [3563], 'deletion': [3567], 'commonly': [3570], 'occurs': [3571], 'exons': [3573], '47–63.25Kinali': [3574], 'Cirak': [3580, 3939], 'Hunt': [3582, 3941], 'Adkin': [3584, 3943], 'Ashton': [3588, 3947], 'Nihoyannopoulos': [3592, 3951], 'al.Local': [3595, 3954], 'AVI-4658': [3604, 3963], 'single-blind,': [3610, 3969], 'placebo-controlled,': [3611, 3970], 'dose-escalation,': [3612, 3971], 'proof-of-concept': [3613, 3972], 'study.Lancet': [3614, 3973], '2009;': [3616, 3975], '8:': [3617, 3976], '918-928Abstract': [3618, 3977], '(571)': [3626, 3985], 'Eteplirsen,': [3629], 'splice-donor': [3633], '51,': [3637], 'induces': [3638], '51.26Aartsma-Rus': [3642], 'Ferlini': [3644], 'Pasmooij': [3648], 'Bushby': [3652], 'Vroom': [3654], 'Balabanov': [3656], 'Translational': [3658], 'challenges': [3661], 'therapies.Hum.': [3665], '2014;': [3668], '25:': [3669], '885-892Crossref': [3670], '(38)': [3673], 'produces': [3677], 'truncated,': [3679], 'in-frame,': [3681], 'partially': [3682], 'functional': [3683, 4052], 'what': [3688], "Becker's": [3692, 3722], 'dystrophy.': [3694], 'variant': [3696], 'leads': [3697], "Duchenne's.": [3716], 'onset': [3720], 'variable': [3725], 'many': [3727, 3741], 'sufferers': [3728], 'retain': [3729], 'ambulate': [3734], 'active': [3737, 4161], 'life': [3738], 'style': [3739], 'decades.': [3742], '2013,': [3745], 'vivo': [3751], 'demonstrated': [3754], 'via': [3759], 'exon-skipping': [3761], 'strategy.21Mendell': [3762], '23Cirak': [3830], '24Mendell': [3887], '25Kinali': [3933], 'study24Mendell': [3993], 'published': [4040], 'eteplirsen.': [4049], 'primary': [4051, 4070], 'assessment': [4053], 'examined': [4054], '6-minute': [4060], 'test': [4062, 4190], '(6MWT).': [4063], 'sole': [4069], 'validated': [4071], 'endpoint': [4073], 'accepted': [4074], 'authorities.22van': [4077], 'Treatments': [4105], 'double': [4107], 'blinded;': [4108], 'randomly': [4111], 'cohorts': [4115], '4': [4118], 'each):': [4119], '(1)': [4120], 'placebo;': [4121], '(2)': [4122], 'mg/kg;': [4125], '(3)': [4127], '50': [4128, 4149], 'mg/kg': [4129], '24': [4133], 'weeks.': [4134], 'then': [4136], 'open-label': [4138, 4145], 'drug,': [4139], 'while': [4140], '(30': [4147], 'mg/kg)': [4150], '3': [4153, 4227], 'years.': [4154], 'treatment,': [4163], 'pooled': [4164], 'controls': [4166, 4202], 'basis': [4171], 'comparison.': [4173], 'extraordinary': [4175], 'provocative': [4177], 'distance': [4185], '6MWT': [4189], '36': [4192], 'months': [4193], '151': [4195], 'm': [4196, 4211, 4221], '0.01;': [4205], '350': [4208, 4218], '250': [4210], 'eteplirsen-treated': [4214, 4233], '100': [4220, 4304], 'controls).': [4225], '16.7%': [4230], 'ambulation,': [4236], '46.2%': [4239], 'Several': [4245], 'treating': [4246, 4299], 'physicians': [4247], 'privately': [4249], 'voiced': [4250], 'substantially': [4255], 'altered': [4256], 'natural': [4258], 'history': [4259], 'responding': [4264], 'addition,': [4267], 'reports': [4271], 'treatment-related': [4273], 'events.': [4276], 'None': [4277], 'interruptions': [4286], 'adjustments.': [4289], 'despite': [4291], 'chorus': [4293], 'eteplersen': [4296], 'advocates,': [4297], 'including': [4298], 'physicians,': [4300], 'parents,': [4301], 'members': [4305], 'Congress,': [4307], 'advisory': [4310], 'panel,': [4311], '6–7': [4314], 'vote,': [4315], "wasn't": [4316], 'convinced:': [4317], 'Sarepta': [4318], 'disregarded': [4320], 'r': [4321]}, 'cited_by_api_url': 'https://api.openalex.org/works?filter=cites:W2600519390', 'counts_by_year': [{'year': 2024, 'cited_by_count': 30}, {'year': 2023, 'cited_by_count': 44}, {'year': 2022, 'cited_by_count': 59}, {'year': 2021, 'cited_by_count': 82}, {'year': 2020, 'cited_by_count': 120}, {'year': 2019, 'cited_by_count': 116}, {'year': 2018, 'cited_by_count': 97}, {'year': 2017, 'cited_by_count': 18}, {'year': 2013, 'cited_by_count': 1}], 'updated_date': '2024-12-15T08:02:41.424611', 'created_date': '2017-04-07'}