Publication Information

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984, 1002, 1009, 1012, 1025, 1034, 1044, 1053, 1059, 1086, 1099, 1115, 1157, 1187, 1200, 1210, 1261, 1311, 1322, 1362, 1387, 1411, 1415, 1422, 1433, 1462, 1485, 1494, 1522, 1554, 1608, 1625, 1648, 1652, 1725, 1728, 1737, 1761, 1800, 1834, 1852, 1876, 1917, 1951, 2019, 2045, 2223, 2238, 2249, 2254, 2295, 2297, 2305, 2391, 2441, 2487, 2519, 2575, 2593, 2597, 2620, 2653, 2683, 2692, 2737, 2761, 2791, 2799, 2865, 2908, 2916, 2936, 2971, 3023, 3036, 3055, 3085, 3101, 3119, 3131, 3146, 3198, 3203, 3235, 3249, 3287, 3312, 3349, 3374, 3414, 3423, 3446, 3456, 3463, 3480, 3498, 3524, 3588, 3644, 3682, 3708, 3725, 3776, 3782, 3789, 3824, 3830, 3894, 3901, 3909, 3914, 3953, 3968, 3976, 3983, 3993, 4004, 4048, 4097, 4119], 'firsts:': [106], 'it': [107, 1492, 1540, 2122], 'is': [108, 113, 185, 234, 283, 345, 372, 475, 1456, 1596, 1619, 1637, 1905, 1989, 2064, 2112, 2123, 2144, 2215], 'first': [110, 122, 129, 135, 157, 546, 776], 'drug': [111, 2021, 2104, 2120, 2263, 3111], 'approved': [114, 123, 130, 139, 1915, 2001], 'DMD': [116, 245, 313, 400, 509, 550, 614, 1245, 1269, 1879, 2164, 2200, 2231, 2355, 2481, 3783], 'United': [119, 1966], 'States,': [120], 'splicing,': [127], 'PMO,': [131], 'also': [133, 154, 1557, 2087], 'be': [138, 160, 426, 461, 478, 503, 890, 942, 1146, 1405, 1466, 1628, 1998, 2060, 2129, 2273], 'based': [140, 677, 1181, 1889, 2004, 2022, 2049, 3925], 'very': [142, 362, 1459, 1907], 'limited': [143], 'data.': [144], 'Although': [145, 627, 1809], 'its': [146, 728, 739, 746], 'was': [148, 540, 597, 622, 629, 643, 670, 787, 793, 815, 843, 878, 886, 952, 1106, 1142, 1686, 1695, 1769, 2097, 2338], 'shrouded': [149], 'controversy,': [151, 2178], 'eteplirsen': [152, 764, 1180, 1234, 1262, 1442, 1487, 1594, 1676, 1718, 1749, 1802, 1831, 1904, 1918, 1941, 2012, 2096, 2117, 2184, 2271, 2488], 'could': [153, 2225], 'become': [155, 243, 1542], 'commercial': [162], 'success.': [163], 'brief': [165], 'commentary': [166], 'reviews': [167], 'disease,': [169], 'therapy,': [171], 'clinical': [173, 689, 743, 1217, 1646, 1895, 2053, 2125, 2227, 2699, 4007], 'data,': [174], 'what': [176], "eteplirsen's": [177, 1692, 1887, 1977], 'means': [179, 2029], 'therapeutics': [183, 1983, 2152, 3418], 'field.DMD': [184], 'an': [186, 197, 340, 504, 566, 1021, 1325, 1621, 1774, 2173, 2261, 2311, 2399, 2657, 3109, 3278, 3333], 'X-linked': [187], 'progressive': [188, 267, 387, 2247], 'disease': [189, 275, 1471, 1561, 1633, 1680, 2076, 2197, 2240], 'which': [191, 811, 1694, 2285], 'patients': [192, 246, 314, 401, 551, 574, 592, 634, 664, 709, 825, 1092, 1117, 1141, 1158, 1212, 1246, 1270, 1292, 1316, 1344, 1364, 1545, 1564, 1602, 1920, 1963, 2154, 2232, 2259], 'lose': [193, 248], 'muscle': [194, 294, 304, 311, 852, 861, 1013, 1027, 1036, 3196, 3237, 3301, 3991], 'function': [195, 250, 2250, 3477], 'from': [196, 230, 395, 1023, 1051, 1111, 1118, 1148, 1207, 1314, 1327, 1806, 2115, 2398, 3107, 3357], 'early': [198], 'age,': [199], 'resulting': [200], 'wheelchair': [202], 'dependency': [203], 'generally': [204], 'around': [205, 215], 'age': [207, 217], '12,': [209, 868, 2996, 3494], 'need': [211, 459], 'assisted': [213], 'ventilation': [214], '20,': [219, 2645, 4037], 'premature': [221, 324], 'death': [222, 3683], 'third–fourth': [225], 'decade': [226], 'life.': [228], 'Apart': [229], 'symptomatic': [231], 'care,': [232], 'there': [233, 628, 1685, 2063, 2111, 2214], 'no': [235, 1366, 2065], 'treatment;': [236], 'while': [237, 1266], 'their': [238, 1069, 1495, 1642, 2006, 3222], 'peers': [239], 'gain': [240], 'skills': [241], 'independent,': [244], 'will': [247, 1465, 1667, 1938, 1946, 1968, 2041, 2069, 2193], 'one': [249, 453, 605, 1615, 1797, 1926, 2113, 2179], 'after': [251, 810, 958, 1319, 1593], 'another': [252], 'until': [253], 'they': [254, 260, 1220, 1587, 1752, 1826, 1937], 'rely': [255], 'around-the-clock': [257], 'care': [258], 'before': [259, 1219, 1317], 'die': [261], 'prematurely.': [262], 'Given': [263], 'severe': [265, 711, 1537], 'nature': [268], 'unmet': [271], 'medical': [272], 'need,': [273, 2156], 'clearly': [276], 'qualifies': [277], 'FDA-accelerated': [280], 'pathway.DMD': [282], 'caused': [284], 'mutations': [286, 316, 330, 470, 1249, 1272, 3784], 'dystrophin': [289, 292, 357, 437, 695, 844, 857, 925, 935, 939, 1017, 1045, 1202, 1248, 1350, 1372, 1394, 1416, 1444, 1463, 1477, 1523, 1531, 1569, 1609, 1626, 1892, 2211, 2224], 'gene.': [290], 'Normally,': [291], 'stabilizes': [293], 'fibers': [295, 305, 853, 877, 897, 993, 1014, 1028, 1037, 1049], 'during': [296], 'contraction': [297], 'connecting': [299], 'actin': [301], 'cytoskeleton': [302], 'within': [303, 1096], 'extracellular': [308], 'matrix': [309], 'surrounding': [310, 1716], 'fibers.': [312], 'have': [315, 402, 657, 826, 1256, 1516, 1550, 1558, 1575, 1603, 1668, 1913, 1921, 1970, 2014, 2204], 'disrupt': [318], 'reading': [320, 334, 423, 466, 3835], 'frame': [321, 335, 424, 3836], 'and/or': [322, 2078], 'cause': [323], 'truncation': [325], 'protein': [327, 343], 'translation.': [328], 'Interestingly,': [329], 'maintain': [332], 'allow': [336, 429], 'production': [338, 431], 'internally': [341, 376], 'deleted': [342, 377], 'partially': [346, 434], 'functional,': [347], 'owing': [348], 'fact': [351, 397, 1849], 'crucial': [354], 'domains': [355], 'are': [358, 379, 1159, 1284, 1418, 1501, 1827, 1860, 1995, 2081], 'located': [359], 'at': [360, 866, 1055, 1263, 1301, 1329, 1333, 1396, 1795, 1865, 2513], 'beginning': [363], 'end': [365], 'protein,': [368], 'whereas': [369, 590], 'middle': [371], 'largely': [373], 'redundant.': [374], 'These': [375, 2258], 'dystrophins': [378], 'found': [380, 1592], 'later': [383], 'onset': [384], 'less': [386], 'Becker': [388], 'dystrophy.The': [390], 'exon': [391, 488, 497, 529, 536, 747, 761, 1253, 1276, 1547, 2289, 2914, 2918, 3931], 'skipping': [392, 472, 490, 498, 530, 748, 1255, 1278, 1549, 2290, 2915], 'approach': [393, 2659], 'stems': [394], 'most': [399, 486, 1962], 'theory': [404], 'genetic': [406], 'capacity': [407], 'produce': [409, 998], 'Becker-like': [410], 'dystrophins.': [411], 'Using': [412, 1123, 3978], 'oligonucleotides': [414, 1994, 2079, 2938, 3998], '(AONs)': [415], 'interfere': [417], 'with': [418, 485, 557, 732, 976, 1073, 1107, 1233, 1271, 1286, 1305, 1338, 1400, 1529, 1565, 1579, 2099, 2137, 2246, 2660, 3569, 4042], 'process,': [421], 'can': [425, 1627, 1677, 2180], 'restored': [427], 'functional': [435, 1070, 1289, 3800], 'Becker-type': [436], 'rather': [438], 'than': [439, 562, 612, 638, 833, 1144, 1385, 1563, 1869, 2270], 'nonfunctional': [441], 'DMD-type': [442], 'dystrophin.': [443, 1003], 'Depending': [444], 'mutation': [447], 'present': [448], 'any': [450, 922, 1176, 1506], 'individual': [451], 'patient,': [452], 'or': [454, 648, 819, 1367, 2002, 4061], 'different': [456, 3780], 'exons': [457, 474, 501], 'may': [458, 502, 656, 1404, 1478, 1524, 2203, 2301], 'skipped': [462], 'restore': [464], 'frame.': [467], 'However,': [468, 905, 1151, 1751], 'because': [469, 1493], 'cluster,': [471], 'certain': [473], 'expected': [476, 1147, 1870], 'therapeutic': [479, 660, 1507, 2906, 2922, 3297, 3400, 3930], 'larger': [481], 'groups': [482, 1156], 'notably': [487], '51': [489, 537, 762, 1254, 1277, 1650], 'applying': [491], '13%–14%': [493], 'patients.': [495, 615, 774, 1448, 2201, 2230], 'Ultimately,': [496], 'various': [500], 'effective': [505, 2047, 2262, 2268], 'therapy': [506, 3062, 3270, 3687, 3838], 'patients.After': [510], 'obtaining': [511], 'proof': [512], 'concept': [514], 'patient-derived': [516], 'cell': [517, 2733], 'models': [518, 1515], 'animal': [520], 'models,': [521], 'two': [522, 601, 1090], 'AON': [523], 'chemistries': [524], 'were': [525, 552, 575, 701, 1066, 1167, 1250, 1380, 1753], 'clinically': [526, 649], 'developed': [527, 541, 2274], 'DMD.': [532, 1872], '2′-O-methyl': [534], 'phosphorothioate': [535, 2738], 'skipper': [538, 763], 'drisapersen': [539, 2392], 'Prosensa/GSK/BioMarin.': [543], 'After': [544, 718], 'dose-finding': [547, 777], 'study,': [548, 589, 1242], '12': [549, 798, 824, 1854], 'treated': [553, 829, 1039, 1063, 1091, 1299], 'off': [556], '6': [558, 563, 582, 1137], 'mg/kg': [559, 821], 'years': [564, 835], 'open-label': [567, 2400], 'study.': [568, 2402], 'Eight': [569], '10': [572, 1851], 'ambulant': [573, 1244], 'stable': [576], 'distance': [579, 1134], 'walked': [580, 1135], 'min': [583, 1138], 'duration': [586, 1410], '2': [591, 603], 'lost': [593, 1094], 'ambulation': [594, 1095, 2493], '[2].': [595], 'Drisapersen': [596], 'then': [598, 1770], 'tested': [599, 767], 'phase': [602, 606, 1230], '3': [607, 1097, 1231], 'placebo-controlled': [608], 'trials': [609, 621, 684, 2054], '300': [613], 'primary': [617, 841], 'endpoint': [618, 842], 'these': [620, 909, 1315, 1572, 1599, 1605, 1813, 2298, 2323], '6-min': [624, 1075, 1172], 'walk': [625, 636, 1076, 1173], 'test.': [626], 'tendency': [631], 'drisapersen-treated': [633], 'further': [637], 'placebo-treated': [639], 'difference': [642], 'small': [644, 1000, 1155, 1185, 1460, 1499, 2026, 2221], 'not': [646, 674, 1168, 1273, 1280, 1342, 1453, 1502, 1576, 1659, 1816, 1939, 1969, 2088], 'statistically': [647], 'significant.Post': [650], 'hoc': [651, 2313], 'analysis': [652, 1310, 4094], 'suggested': [653, 989], 'treatment': [655, 1323, 1595, 1942, 1945, 2192, 2440, 3707], 'had': [658, 1226, 1297, 1365, 1604, 1745, 1881], 'effect': [661, 2208, 2486], 'younger': [663], 'marketing': [667, 729], 'application': [669, 731, 1488, 2105, 2661], 'filed.': [671], 'FDA': [672, 720, 915, 937, 1165, 1191, 1438, 1483, 1704, 1765, 1778, 2032, 2068, 2139], 'did': [673], 'grant': [675], 'this,': [676], 'facts': [680], 'pivotal': [683], 'failed': [685], 'demonstrate': [687], 'significant': [688, 699], 'benefit': [690, 1508, 1647], '(or': [691], 'clear': [692, 1543], 'increase': [693, 923, 1022, 1326, 1370, 1622], 'expression)': [696], 'toxicities': [700], 'observed': [702, 1143], '(skin': [703], 'reactions': [704], 'proteinuria': [706], 'many': [708], 'thrombocytopenia': [712], 'about': [714, 1360], '2%': [715], 'patients).': [717], 'rejection': [721], "drisapersen's": [723], 'application,': [724], 'BioMarin': [725, 2329], 'recently': [726], 'withdrew': [727], 'authorization': [730], 'European': [734], 'Medicine': [735], 'Agency': [736], 'announced': [738, 2106], 'decision': [740, 1788], 'stop': [742], 'development': [744, 2126, 2880], 'compounds': [749, 758], 'focus': [752], 'next': [755, 2256], 'generation': [756], '[3].The': [759], 'PMO': [760], 'has': [765, 1541, 1658, 1815, 2033, 2286, 2345], 'been': [766, 828, 1257, 1298, 1577, 1660, 1817, 2346], 'far': [770], 'smaller': [771], 'cohort': [772], 'A': [775, 2609, 2651, 2679, 3466, 4019], 'study': [778, 792, 1296], 'revealed': [779, 964, 1324, 1517], 'highest': [782], 'dose': [783], 'tested,': [784], '20': [785], 'mg/kg,': [786, 803, 1265], 'suboptimal.': [788], 'Then': [789], 'new': [791, 1215, 2103, 2151, 2757, 2900], 'initiated': [794, 1227], '2011': [796], 'comparing': [800], '30,': [801], '50': [802, 820], 'placebo': [805, 813, 1081], 'weekly': [806, 830, 1259], '24': [808, 1084], 'weeks,': [809, 872], 'group': [814, 1082, 1875], 'switched': [816], '30': [818, 1264], 'dosing.': [822], 'All': [823], 'now': [827, 2042], '4': [834], 'without': [836, 1960], 'apparent': [837, 1369], 'treatment-related': [838], 'side': [839, 3408], 'effects.The': [840], 'restoration': [845, 933, 1417], 'as': [846, 995, 1126, 1843, 2172, 2341, 2812, 2920, 2979, 3583], 'assessed': [847, 1067], 'percentage': [850, 1011], 'express': [855], 'detectable': [856, 1016], 'immunohistochemistry': [859, 1019], 'biopsies.': [862], 'In': [863, 1240, 1358, 1721], 'biopsies': [864, 956, 983, 1064, 1209, 1312, 1403], 'taken': [865, 957, 1313], 'baseline,': [867], '24,': [869], '48': [871, 1303, 1320, 1334, 1397], 'dystrophin-positive': [876, 896], 'counted': [879], 'subjectively': [880], 'blinded': [883], 'manner': [884, 2143], 'initially': [887, 1746], 'reported': [888], 'significantly': [891, 1532, 3817], 'increased': [892, 1050, 1201], '30%–50%': [894, 992], '24-': [900], '48-week-treated': [902], 'specimens': [903], '[4].': [904], 'subsequent': [906], 'reanalysis': [907], 'biopsy': [910], 'slides': [911], 'performed': [912, 953], 'raised': [916], 'questions': [917], 'regarding': [918], 'magnitude': [920], 'expression': [926, 1046, 1203, 1373, 1395, 1464, 3486], '[5].To': [927], 'rigorously': [929, 1006], 'evaluate': [930], 'possible': [932, 1105], 'expression,': [936, 2212], 'requested': [938], 'amounts': [940, 1001, 1607, 2222], 'quantified': [943, 1578], 'well-controlled': [946], 'western': [947, 1205, 1308, 1354, 1582], 'blot': [948, 1206, 1309, 1355, 1583], 'assay,': [949], 'additional': [955, 1208], '188': [959], 'weeks': [960, 1085, 1304, 1321, 1335, 1398], 'treatment.': [962], 'average': [965], 'levels': [966, 1414, 1445, 1553, 1570, 2592], '0.93%': [968, 1425], 'normal': [971, 1054, 1060, 1388], 'amount': [972, 1461], 'dystrophin,': [974, 1555], 'compared': [975, 1399], 'baseline': [978, 1056, 1330, 1552], 'value': [979], '0.08%': [981], 'untreated': [985, 1030], 'muscles.': [987], 'designated': [994], 'positive': [996, 1048], 'each': [997], 'only': [999, 1103, 2181, 2205], 'Indeed,': [1004, 1873], 'controlled': [1007], 'assessment': [1008], 'expressing': [1015], 'showed': [1020], '1.1%': [1024], 'dystrophin-expressing': [1026], 'samples': [1031], '17.4%': [1033], 'biopsies,': [1040], 'intensity': [1043], '9.4%': [1052], '22.6%': [1058], '[6].Patients': [1065], 'status': [1071], 'primarily': [1072], 'test,': [1077], 'but': [1078, 1283, 1925, 2213], 'lacking': [1079], 'beyond': [1083], 'treatment,': [1087, 1282], 'having': [1089], 'who': [1093, 1783], 'months': [1098], 'trial': [1100, 1218, 1232, 1859], 'initiation,': [1101], 'comparison': [1104], 'historical': [1108, 1124, 2490], 'controls': [1109, 1125], 'selected': [1110], 'natural': [1112, 1149], 'history': [1113], 'data': [1114, 1451, 1893, 2027], 'baseline-matched': [1116], 'Belgium': [1119], 'Italy': [1121], '[7].': [1122], 'comparison,': [1128], 'slower': [1130, 1560, 2196], 'decrease': [1131], 'eteplirsen-treated': [1140, 1211], 'would': [1145, 1221, 2059], 'history.': [1150], 'such': [1152, 1183, 1498, 2024], 'comparisons': [1153], 'challenging': [1160], 'interpret,': [1162], 'reviewers': [1166, 1439, 1484], 'convinced': [1169], 'times': [1174], 'demonstrated': [1175, 1345], 'benefit.Reluctant': [1177], 'approve': [1179], 'patient': [1188, 1838, 1956, 2165], 'samples,': [1189], 'asked': [1192], 'Sarepta': [1193, 1225, 1666, 2515], 'June': [1195, 2839, 3432, 3619, 3667, 3949], 'provide': [1198, 1427, 1505, 2226], 'evidence': [1199, 1428], 'ongoing': [1216, 2160], 'make': [1222], 'decision.': [1224], 'confirmatory': [1229, 1671], '(PROMOVI)': [1235], '2014': [1238], '[8].': [1239], '80': [1243, 1267], 'whose': [1247], 'amenable': [1251, 1274, 3256], 'receiving': [1258, 1923], 'dosing': [1260], 'matched': [1268], 'do': [1279, 1426, 1452], 'receive': [1281, 2302], 'followed': [1285], 'same': [1288], 'outcome': [1290, 2700], 'measures.Thirteen': [1291], 'PROMOVI': [1295], 'least': [1302, 1796], 'eteplirsen,': [1306], '0.16%': [1328], '0.44%': [1332], 'average,': [1337], 'caveat': [1340], 'all': [1343, 2176], 'post-treatment': [1347], 'response': [1348], "Sarepta's": [1352, 2342], 'validated': [1353, 1581], 'system': [1356], '[9,10].': [1357], 'fact,': [1359], 'half': [1361], 'minimal': [1368], 'increases': [1376, 1392, 1421, 1443, 1475, 1500, 1591], 'others': [1379], 'higher,': [1381], 'up': [1382], '1%': [1386], '[10].': [1389, 1720, 1791], 'lower': [1391], '180-week': [1402], 'related': [1406, 1828], 'longer': [1409, 2190], 'therapy.Although': [1412], 'disappointing,': [1419], '0.28%': [1423], 'intended': [1431], 'mechanism': [1432, 2758], 'action': [1434], 'eteplirsen:': [1436], 'agreed': [1440], 'some': [1447, 1482, 1899, 2296], 'What': [1449], 'tell': [1454], 'us': [1455], 'whether': [1457, 1620], 'enough': [1467], 'slow': [1469, 1631, 1678], 'down': [1470, 1632, 1679], 'progression.': [1472, 1681], 'Such': [1473], 'modest': [1474, 2207], 'seem': [1479], 'negligible,': [1480], 'wanted': [1489], 'reject': [1491], 'beliefs': [1496], 'going': [1503, 1996], '[10].Nevertheless,': [1509], 'earlier': [1510], 'preclinical': [1511], 'studies': [1512, 1672], 'mouse': [1514, 3522], 'little': [1520], 'bit': [1521], 'go': [1525, 2334], 'long': [1527], 'way,': [1528], '<4%': [1530], 'increasing': [1533], 'survival': [1534], 'background.': [1538], 'Furthermore,': [1539, 1598], 'requiring': [1546], '44': [1548], 'higher': [1551, 1868], 'progression': [1562, 1634, 2198], 'other': [1566, 1798, 1992, 2074], 'mutations.': [1567], 'however,': [1574], 'system,': [1584], 'so': [1585], 'how': [1586], 'relate': [1588], 'unclear.': [1597], 'mice': [1600], 'low': [1606], 'since': [1610], 'birth.': [1611], 'question': [1613], 'cannot': [1616], 'answer': [1617], 'yet': [1618], '0.28%–0.93%': [1624], 'sufficient': [1629], 'once': [1635], 'pathology': [1636, 3519], 'established.FDA': [1638], 'indeed': [1639, 2185], 'stated': [1640], '"A': [1645], 'Exondys': [1649], '(tradename': [1651], 'eterplirsen),': [1653], 'including': [1654], 'improved': [1655], 'motor': [1656], 'function,': [1657], 'established"': [1661], '[1]': [1662], 'mandated': [1664], 'perform': [1670], 'establish': [1674], 'Within': [1682], 'FDA,': [1684], 'intense': [1687], 'unprecedented': [1689], 'disagreement': [1690], 'concerning': [1691], 'approval,': [1693], 'made': [1696, 1818], 'public': [1697], '16,': [1700, 3304], '2016,': [1701, 1935, 2525], 'when': [1702], 'commissioner': [1705], 'released': [1706], 'document': [1708], 'summarizing': [1709], 'remarkable': [1711], 'detail': [1712], 'internal': [1714, 1764], 'controversies': [1715], 'review': [1719, 1803, 2652], 'brief,': [1722], 'Director': [1724, 1760], 'Office': [1727], 'Evaluation,': [1730], 'Dr.': [1731, 1742, 1756, 1780, 1785, 1792], 'Ellis': [1732], 'Unger,': [1733], 'Division': [1736], 'Neurology': [1738], 'Products,': [1739], 'headed': [1740], 'Ronald': [1743], 'Farkas,': [1744], 'rejected': [1747, 2003], 'application.': [1750], 'overruled': [1754], 'Janet': [1757], 'Woodcock,': [1758], 'CDER.': [1762], 'An': [1763, 2844], 'dispute': [1766], 'resolution': [1767], 'process': [1768], 'initiated,': [1771], 'culminating': [1772], 'appeal': [1775], 'Commissioner,': [1779], 'Robert': [1781], 'Califf,': [1782], 'allowed': [1784], "Woodcock's": [1786], 'stand': [1790], 'Farkas': [1793], 'member': [1799], 'team': [1804], 'resigned': [1805], 'FDA.': [1808], 'reason': [1811, 2216], 'resignations': [1814], 'public,': [1819], 'timing': [1821], 'led': [1822], 'speculation': [1824], 'approval.': [1832], 'Regardless': [1833], 'controversies,': [1836], 'community': [1839], 'celebrates': [1840], 'victory,': [1845], 'pointing': [1846], 'boys': [1855], 'original': [1858], 'still': [1861], 'walking': [1862], 'functioning': [1864], 'level': [1867], '36': [1877], 'prominent': [1878], 'clinician-scientists': [1880], 'published': [1882], 'letter': [1884], 'calling': [1885], 'results': [1896, 2397], '[11].': [1897], 'Meanwhile,': [1898], 'critical': [1900], 'voices': [1901], 'argue': [1902], 'expensive': [1908], 'placebo.': [1909], 'Several': [1910], 'insurance': [1911, 1928], 'companies': [1912, 2040, 2350], 'already': [1914, 2107], 'coverage': [1916], 'begun': [1922], 'it,': [1924], 'major': [1927], 'company,': [1929], 'Anthem,': [1930], 'indicated': [1931], 'October': [1933, 2524, 2823, 3074, 3389, 3557, 3844], '7,': [1934], 'reimburse': [1940], '[12].': [1943], 'cost': [1947], 'order': [1950], '$300,000–400,000': [1952], 'per': [1953, 1955], 'year': [1954], 'initially,': [1957], 'meaning': [1958], 'reimbursement,': [1961], 'States': [1967], 'access': [1971], 'drug.What': [1974], 'lessons': [1975], 'does': [1976, 2186], 'hold': [1979], 'community?': [1984], 'One': [1985, 2085, 2404], 'point': [1986], 'worth': [1987], 'noting': [1988], 'that,': [1990], 'like': [1991], 'drugs,': [1993], 'reviewed': [1999], 'merits.': [2007], 'Some': [2008], 'commentators': [2009], 'controversy': [2013], 'expressed': [2015], 'fears': [2016], 'set': [2028], '"lowered': [2034], 'bar"': [2036, 2072], 'seek': [2043], 'marginally': [2046], 'drugs': [2048, 2077, 2269, 3705], 'small,': [2051], 'underpowered': [2052], 'unvalidated': [2056], 'endpoints.': [2057], 'That': [2058], 'mistake,': [2062], 'indication': [2066], '"lower': [2070], 'rare': [2075, 3114, 3709], 'currently': [2082], 'development.': [2084], 'should': [2086, 2128], 'forget': [2089], 'road': [2092], 'long,': [2098], 'plans': [2100], '2013': [2109], '[13].If': [2110], 'lesson': [2114], 'saga': [2118], 'developers,': [2121], 'always': [2127], 'rigorous': [2130], 'well': [2132], 'controlled,': [2133], 'working': [2136, 2351], 'constructive': [2142], 'best': [2146], 'way': [2147], 'rapidly': [2149], 'deliver': [2150], 'perhaps': [2157], 'using': [2158, 3332], 'dialogue': [2161], 'between': [2162], 'community,': [2166], 'academics,': [2167], 'regulators': [2169], 'Europe': [2171], 'example': [2174, 2574], '[14].In': [2175], 'hope': [2182, 2265], 'work': [2187], 'term': [2191], 'reveal': [2194], 'restoring': [2210, 3837], 'expect': [2218], 'even': [2220], 'benefits': [2228], 'parents': [2234], 'face': [2235], 'consequences': [2237], 'daily': [2243], 'basis,': [2244], 'coping': [2245], 'loss': [2248, 2253], 'dreading': [2252], 'function.': [2257], 'deserve': [2260], 'future.Author': [2277], 'Disclosure': [2278], 'StatementA.A.R.': [2279], 'report': [2280], 'being': [2281, 2310], 'employed': [2282, 2340], 'LUMC,': [2284], 'patents': [2287], 'technology.': [2291], 'As': [2292], 'coinventor': [2294], 'patents,': [2299], 'A.A.R.': [2300, 2308], 'share': [2304], 'potential': [2306, 2905], 'royalties.': [2307], 'reports': [2309], 'ad': [2312], 'consultant': [2314, 2348], 'BioMarin,': [2316], 'PTC,': [2317], 'BMS,': [2318], 'Summit.': [2320], 'Remuneration': [2321], 'activities': [2324, 3328], 'speaker': [2326], 'honoraria': [2327], 'PTC-organized': [2331], 'satellite': [2332, 2517], 'symposia': [2333], 'LUMC.': [2336], 'A.M.K.': [2337], 'previously': [2339], 'CSO': [2343], 'RNA': [2353, 2894, 3045, 3155, 3415, 3511], 'therapeutics.References1': [2356], 'www.fda.gov/NewsEvents/Newsroom/PressAnnouncements/ucm521263.htm': [2357], 'Google': [2358, 2408, 2411, 2450, 2453, 2456, 2503, 2506, 2528, 2531, 2534, 2537, 2540, 2584], 'Scholar2': [2359], 'Goemans': [2360, 2460, 2550], 'NM,': [2361], 'Tulinius': [2362], 'M,': [2363, 2367, 2549], 'van': [2364, 2374], 'den': [2365], 'Hauwe': [2366], 'Kroksmark': [2368], 'AK,': [2369], 'Buyse': [2370], 'G,': [2371, 2560], 'Wilson': [2372], 'RJ,': [2373], 'Deutekom': [2375], 'JC,': [2376], 'de': [2377], 'Kimpe': [2378], 'SJ,': [2379], 'Lourbakos': [2380], 'A,': [2381, 2554, 2556], 'Campion': [2383, 2559], 'G.': [2384], '(2016).': [2385, 2563], 'Long-term': [2386], 'efficacy,': [2387], 'safety,': [2388], 'pharmacokinetics': [2390], 'dystrophy:': [2396], 'extension': [2401], 'PLoS': [2403], '11:e0161955.': [2405], 'Crossref,': [2406, 2448, 2501, 2582], 'Medline,': [2407, 2449, 2502, 2583], 'Scholar3': [2409], 'http://investors.bmrn.com/releasedetail.cfm?ReleaseID=973536': [2410], 'Scholar4': [2412], 'Mendell': [2413, 2458], 'JR,': [2414, 2459], 'Rodino-Klapac': [2415], 'LR,': [2416], 'Sahenk': [2417], 'Z,': [2418], 'Roush': [2419], 'K,': [2420, 2467, 2547], 'Bird': [2421], 'L,': [2422, 2426], 'Lowes': [2423, 2462], 'LP,': [2424, 2463], 'Alfano': [2425, 2464], 'Gomez': [2427], 'AM,': [2428], 'Lewis': [2429], 'S,': [2430], 'et': [2431, 2561], 'al.;': [2432], 'Study': [2434, 2476, 2611, 3127], 'Group.': [2435], '(2013).': [2436], 'dystrophy.': [2444, 2497, 2578], 'Ann': [2445, 2498], 'Neurol': [2446, 2499, 2580], '74:637–647.': [2447], 'Scholar5': [2451], 'www.fda.gov/downloads/advisorycommittees/committeesmeetingmaterials/drugs/peripheralandcentralnervoussystemdrugsadvisorycommittee/ucm481912.pdf': [2452], 'Scholar6': [2454], 'www.fda.gov/downloads/advisorycommittees/committeesmeetingmaterials/drugs/peripheralandcentralnervoussystemdrugsadvisorycommittee/ucm497064.pdf': [2455], 'Scholar7': [2457], 'N,': [2461, 2551], 'LN,': [2465], 'Berry': [2466], 'Shao': [2468], 'J,': [2469], 'Kaye': [2470], 'EM,': [2471], 'Mercuri': [2473], 'E;': [2474], 'Group': [2477], 'Telethon': [2479], 'Foundation': [2480], 'Italian': [2482], 'Network.': [2483], '2016).': [2484], 'Longitudinal': [2485], 'versus': [2489], 'control': [2491], '79:257–271.': [2500], 'Scholar8': [2504], 'https://clinicaltrials.gov/ct2/show/NCT02255552': [2505], 'Scholar9': [2507], 'Presented': [2508], 'Prof.': [2510], 'Francesco': [2511], 'Muntoni': [2512], 'sponsored': [2516], 'symposium': [2518], 'World': [2521], 'Muscle': [2522, 2798, 4024], 'Society,': [2523], 'Granada,': [2526], 'Spain': [2527], 'Scholar10': [2529], 'www.accessdata.fda.gov/drugsatfda_docs/nda/2016/206488_summary%20review_Redacted.pdf': [2530], 'Scholar11': [2532], 'www.cdmd.ucla.edu/files/view/FDA_ETEPLIRSEN_LETTER_02242016.pdf': [2533], 'Scholar12': [2535], 'www.srnnews.com/anthem-says-will-not-cover-first-fda-approved-duchenne-drug': [2536], 'Scholar13': [2538], 'www.drugs.com/nda/eteplirsen_130725.html': [2539], 'Scholar14': [2541], 'Straub': [2542], 'V,': [2543], 'Balabanov': [2544], 'P,': [2545], 'Bushby': [2546], 'Ensini': [2548], 'De': [2552], 'Luca': [2553], 'Pereda': [2555], 'Hemmings': [2557], 'R,': [2558], 'al.': [2562], 'Stakeholder': [2564], 'cooperation': [2565], 'overcome': [2567], 'challenges': [2568, 3069, 3413, 3944], 'orphan': [2570], 'medicine': [2571, 2719], 'development:': [2572], 'Lancet': [2579], '15:882–890.': [2581], 'ScholarFiguresReferencesRelatedDetailsCited': [2585], 'byTherapeutic': [2586], 'strategies': [2587, 3216, 3298], 'autism:': [2589], 'targeting': [2590, 2625, 3254], 'three': [2591], 'central': [2595], 'dogma': [2596], 'molecular': [2598], 'biology16': [2599], 'February': [2600, 3260, 3452, 3981], '2023': [2601], '|': [2602, 2641, 2669, 2708, 2743, 2765, 2805, 2825, 2841, 2857, 2874, 2888, 2949, 2968, 2993, 3009, 3052, 3076, 3144, 3168, 3189, 3209, 3241, 3262, 3285, 3339, 3367, 3391, 3434, 3454, 3470, 3490, 3510, 3536, 3559, 3574, 3621, 3669, 3695, 3766, 3846, 3876, 3898, 3935, 3951, 4017, 4080, 4107, 4126], 'Translational': [2603], 'Psychiatry,': [2604], 'Vol.': [2605, 2630, 2644, 2673, 2711, 2726, 2746, 2768, 2787, 2807, 2830, 2847, 2859, 2876, 2892, 2910, 2931, 2953, 2974, 2995, 3013, 3039, 3058, 3079, 3151, 3170, 3191, 3213, 3230, 3244, 3265, 3290, 3303, 3315, 3341, 3352, 3371, 3393, 3405, 3425, 3437, 3459, 3472, 3493, 3513, 3540, 3561, 3577, 3625, 3673, 3701, 3714, 3770, 3793, 3810, 3832, 3852, 3882, 3904, 3920, 3939, 3957, 3996, 4012, 4036, 4058, 4084, 4111], '13,': [2606, 2747, 2808, 3192], '1PPMOs:': [2608], 'Case': [2610], 'Cell‐Penetrating': [2613], 'Peptide': [2614], 'Application2': [2615], 'December': [2616, 2639, 3283], '2022Selection': [2617], 'identification': [2619], 'novel': [2622], 'ssDNA': [2623], 'aptamer': [2624], 'human': [2626, 2776, 3072, 3450], 'skeletal': [2627, 3195, 3236, 3300, 3805, 4055], 'muscleBioactive': [2628], 'Materials,': [2629], '20A': [2631], 'Multifunctional': [2632], 'Porous': [2633], 'Silicon': [2634], 'Nanocarrier': [2635], 'Glioblastoma': [2637], 'Treatment9': [2638], '2022': [2640, 2668, 2707, 2742, 2764, 2804, 2840, 2856, 2873, 2887, 2967, 2992, 3008, 3051, 3143, 3167, 3188, 3208], 'Molecular': [2642, 2766, 2828, 2972, 3056, 3696, 3880], 'Pharmaceutics,': [2643, 3831], '1Reproductive': [2647], 'sterility': [2648], 'aquaculture:': [2650], 'induction': [2654], 'methods': [2655], 'emerging': [2658], 'Pacific': [2663], 'Northwest': [2664], 'finfish': [2665], 'species20': [2666], 'July': [2667, 2741, 2872, 2886], 'Reviews': [2670], 'Aquaculture,': [2672], '15,': [2674, 2877], '1Mesyl': [2676], 'Phosphoramidate': [2677, 3581], 'Oligonucleotides:': [2678, 3886, 3911], 'New': [2680, 3887, 3889, 3892], 'Promising': [2681, 2981], 'Type': [2682], 'Antisense': [2684, 3379, 3634], 'Agents19': [2685], 'January': [2686, 3091, 3166, 3187, 3239, 3508, 3572, 3933], '2023Peptide-Conjugated': [2687], 'PMOs': [2688, 3086], 'Treatment': [2691, 3133, 3962], 'Myotonic': [2693], 'Dystrophy19': [2694], 'November': [2695, 2803, 3337, 3365, 4124], '2022Therapeutic': [2696], 'opportunities': [2697, 2716], 'measures': [2701], 'dystrophy24': [2705], 'May': [2706, 2763], 'Neurological': [2709, 3164, 3185, 3791], 'Sciences,': [2710, 2973, 3057, 3792], '43,': [2712], 'S2Challenges': [2714], 'precision': [2718], 'neurodevelopmental': [2721], 'disordersAdvanced': [2722], 'Delivery': [2724, 3084, 3403, 4027], 'Reviews,': [2725, 3404, 3576], '191An': [2727], 'LNA-amide': [2728], 'modification': [2729], 'enhances': [2731, 2933], 'uptake': [2734], 'activity': [2736, 3823], 'exon-skipping': [2739, 3258], 'oligonucleotides12': [2740], 'Nature': [2744], 'Communications,': [2745, 4110], '1Human': [2749], 'Identical': [2750], 'Sequences,': [2751], 'hyaluronan,': [2752], 'hymecromone': [2754], '─': [2755], 'management': [2760], 'COVID-1920': [2762], 'Biomedicine,': [2767], '3,': [2769], '1Harnessing': [2771], 'nucleic': [2772], 'acid': [2773, 3397, 3795], 'technologies': [2774], 'health': [2777], 'earth': [2779], 'spaceLife': [2782], 'Sciences': [2783], 'Space': [2785], 'Research,': [2786, 3212], '35Strategies': [2788], 'Bottlenecks': [2790], 'rAAV-Mediated': [2792], 'Expression': [2793], 'Skeletal': [2795], 'Cardiac': [2797], 'Dystrophy3': [2802], 'Genes,': [2806, 3190], '11Nucleic': [2810, 3396], 'Acids': [2811, 2868], 'Novel': [2813], 'Therapeutic': [2814, 2870, 2982, 3175, 3552], 'Modalities': [2815], 'Address': [2817], 'Multiple': [2818], 'Sclerosis': [2819], 'Onset': [2820], 'Progression25': [2822], '2021': [2824, 2948, 3240, 3261, 3284, 3338, 3366, 3390, 3433, 3453, 3469, 3489, 3509, 3558, 3573, 3620, 3668, 3694, 3765, 3875, 3897, 3934], 'Cellular': [2826, 3457], 'Neurobiology,': [2829], '42,': [2831, 3541], '8Emerging': [2833], 'Approaches': [2834], 'Enabling': [2836], 'RNAi': [2837], 'Therapeutics30': [2838], 'Chemistry': [2842, 2864], '–': [2843], 'Asian': [2845], 'Journal,': [2846], '17,': [2848, 3940], '16Meeting': [2850], 'report:': [2851], 'ADME': [2853], 'workshop12': [2854], 'Xenobiotica,': [2858], '52,': [2860], '8The': [2862], 'Medicinal': [2863, 4034], 'Artificial': [2866], 'Oligonucleotides22': [2871], '8Drug': [2879], 'progress': [2881], 'duchenne': [2883, 3250], 'dystrophy22': [2885], 'Frontiers': [2889, 3010, 3368], 'Pharmacology,': [2891, 3370], '13Alternative': [2893], 'defects': [2896], 'pediatric': [2898], 'cancers:': [2899], 'insights': [2901], 'tumorigenesis': [2903], 'vulnerabilitiesAnnals': [2907], 'Oncology,': [2909], '33,': [2911], '6Targeted': [2913], 'NF1': [2917], '17': [2919], 'neurofibromatosis': [2924], 'type': [2925], 'IMolecular': [2926], 'Therapy': [2927, 3156, 3307], '-': [2928, 4074], 'Acids,': [2930], '28Radiation': [2932], 'delivery': [2935, 3398, 3803, 3989, 4047], 'improves': [2940], 'chemo-radiation': [2941], 'efficacy': [2942], 'brain': [2944], 'tumor': [2945, 3443], 'xenografts14': [2946], 'April': [2947, 2966, 2991, 3488, 3896, 4105], 'Cancer': [2950, 4120], 'Gene': [2951, 3077, 3427, 3642, 4030], 'Therapy,': [2952, 3078, 3809, 3851, 3919], '29,': [2954, 3080, 3811, 3921], '5Advanced': [2956], 'Gene-Targeting': [2957], 'Therapies': [2958], 'Motor': [2960], 'Neuron': [2961], 'Diseases': [2962], 'Dystrophies27': [2965], 'International': [2969, 3053, 4127], 'Journal': [2970, 3054, 3145, 3286, 3455, 3829, 3900, 3952, 4128], '23,': [2975, 3059], '9Splicing': [2977], 'Modulation': [2978], 'Strategy': [2983], 'Lysosomal': [2985], 'Storage': [2986], 'Disorders:': [2987], 'Mucopolysaccharidoses': [2989], 'Example19': [2990], 'Life,': [2994], '5Therapeutic': [2998], 'Strategies': [2999], 'Dystrophin': [3001, 3977], 'Replacement': [3002], 'Dystrophy28': [3006], 'March': [3007, 3050, 3142, 3693, 3764, 3874, 4078], 'Medicine,': [3012, 3424, 3700], '9Stride': [3014], 'Velocity': [3015], '95th': [3016], 'Centile:': [3017], 'Insights': [3018], 'into': [3019], 'Gaining': [3020], 'Regulatory': [3021], 'Qualification': [3022], 'First': [3025], 'Wearable-Derived': [3026], 'Digital': [3027], 'Endpoint': [3028], 'use': [3030, 4005], 'Dystrophy': [3034, 3863, 3971], 'TrialsJournal': [3035], 'Neuromuscular': [3037, 3313, 3347, 3350], 'Diseases,': [3038, 3314, 3351], '9,': [3040, 3702], '2Current': [3042], 'Advances': [3043, 3153, 3429], 'Therapeutics': [3046, 3122, 3344], 'Human': [3048], 'Diseases1': [3049], '5Gene': [3061], 'gene': [3064, 3485, 3506, 3686], 'correction:': [3065], 'targets,': [3066], 'progress,': [3067], 'treating': [3071], 'diseases9': [3073], '2020': [3075, 3535, 3845, 3950, 4079, 4106, 4125], '1-2In': [3082], 'Vitro': [3083], 'Myoblasts': [3088], 'Electroporation1': [3090], '2022Polypharmacology': [3092], 'Design': [3095, 3100], 'Discovery—Basis': [3097], 'Rational': [3099], 'Multitarget': [3102], 'Drugs2': [3103], 'August': [3104], '2022Lessons': [3105], 'learned': [3106], 'developing': [3108], 'diseaseCMC': [3115], 'regulatory': [3117], 'aspects': [3118], 'therapeuticsTargeted': [3121], 'Rare': [3124, 3346], 'DisordersA': [3125], 'Long-Term': [3126], 'Evaluating': [3128], 'Effects': [3130], 'Nicorandil': [3132], 'Dystrophy-Associated': [3137], 'Cardiomyopathy': [3138], 'mdx': [3140], 'Mice30': [3141, 3388], 'Cardiovascular': [3147], 'Pharmacology': [3148], 'Therapeutics,': [3150, 3436, 3624, 3672, 3769], '27Recent': [3152], 'Its': [3158], 'Carriers': [3159], 'Treat': [3161], 'Single-Gene': [3163], 'Disorders12': [3165], 'Biomedicines,': [3169], '10,': [3171, 4013], '1Nucleic': [3173], 'Acid-Based': [3174], 'Approach': [3176, 3553], 'Spinal': [3178], 'Bulbar': [3180], 'Atrophy': [3182], 'Related': [3184], 'Disorders5': 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