Publication Information

{'id': 'https://openalex.org/W2560408974', 'doi': 'https://doi.org/10.1038/mt.2016.188', 'title': 'Eteplirsen Approved for Duchenne Muscular Dystrophy: The FDA Faces a Difficult Choice', 'display_name': 'Eteplirsen Approved for Duchenne Muscular Dystrophy: The FDA Faces a Difficult Choice', 'publication_year': 2016, 'publication_date': '2016-11-01', 'ids': {'openalex': 'https://openalex.org/W2560408974', 'doi': 'https://doi.org/10.1038/mt.2016.188', 'mag': '2560408974', 'pmid': 'https://pubmed.ncbi.nlm.nih.gov/27916994', 'pmcid': 'https://www.ncbi.nlm.nih.gov/pmc/articles/5154484'}, 'language': 'en', 'primary_location': {'is_oa': True, 'landing_page_url': 'https://doi.org/10.1038/mt.2016.188', 'pdf_url': 'https://www.cell.com/article/S1525001616454390/pdf', 'source': {'id': 'https://openalex.org/S51596929', 'display_name': 'Molecular Therapy', 'issn_l': '1525-0016', 'issn': ['1525-0016', '1525-0024'], 'is_oa': False, 'is_in_doaj': False, 'is_core': True, 'host_organization': 'https://openalex.org/P4310320990', 'host_organization_name': 'Elsevier BV', 'host_organization_lineage': ['https://openalex.org/P4310320990'], 'host_organization_lineage_names': ['Elsevier BV'], 'type': 'journal'}, 'license': 'cc-by-nc-nd', 'license_id': 'https://openalex.org/licenses/cc-by-nc-nd', 'version': 'publishedVersion', 'is_accepted': True, 'is_published': True}, 'type': 'editorial', 'type_crossref': 'journal-article', 'indexed_in': ['crossref', 'pubmed'], 'open_access': {'is_oa': True, 'oa_status': 'hybrid', 'oa_url': 'https://www.cell.com/article/S1525001616454390/pdf', 'any_repository_has_fulltext': True}, 'authorships': [{'author_position': 'first', 'author': {'id': 'https://openalex.org/A5109357631', 'display_name': 'Cy A. Stein', 'orcid': None}, 'institutions': [], 'countries': [], 'is_corresponding': True, 'raw_author_name': 'Cy A Stein', 'raw_affiliation_strings': ['Member, Editorial Board'], 'affiliations': [{'raw_affiliation_string': 'Member, Editorial Board', 'institution_ids': []}]}], 'institution_assertions': [], 'countries_distinct_count': 0, 'institutions_distinct_count': 0, 'corresponding_author_ids': ['https://openalex.org/A5109357631'], 'corresponding_institution_ids': [], 'apc_list': {'value': 3900, 'currency': 'USD', 'value_usd': 3900, 'provenance': 'doaj'}, 'apc_paid': {'value': 3900, 'currency': 'USD', 'value_usd': 3900, 'provenance': 'doaj'}, 'fwci': None, 'has_fulltext': True, 'fulltext_origin': 'ngrams', 'cited_by_count': 65, 'citation_normalized_percentile': {'value': 0.93796, 'is_in_top_1_percent': False, 'is_in_top_10_percent': True}, 'cited_by_percentile_year': {'min': 97, 'max': 98}, 'biblio': {'volume': '24', 'issue': '11', 'first_page': '1884', 'last_page': '1885'}, 'is_retracted': False, 'is_paratext': False, 'primary_topic': {'id': 'https://openalex.org/T10441', 'display_name': 'Muscle Physiology and Disorders', 'score': 0.9997, 'subfield': {'id': 'https://openalex.org/subfields/1312', 'display_name': 'Molecular Biology'}, 'field': {'id': 'https://openalex.org/fields/13', 'display_name': 'Biochemistry, Genetics and Molecular Biology'}, 'domain': {'id': 'https://openalex.org/domains/1', 'display_name': 'Life Sciences'}}, 'topics': [{'id': 'https://openalex.org/T10441', 'display_name': 'Muscle Physiology and Disorders', 'score': 0.9997, 'subfield': {'id': 'https://openalex.org/subfields/1312', 'display_name': 'Molecular Biology'}, 'field': {'id': 'https://openalex.org/fields/13', 'display_name': 'Biochemistry, Genetics and Molecular Biology'}, 'domain': {'id': 'https://openalex.org/domains/1', 'display_name': 'Life Sciences'}}, {'id': 'https://openalex.org/T10878', 'display_name': 'CRISPR and Genetic Engineering', 'score': 0.9939, 'subfield': {'id': 'https://openalex.org/subfields/1312', 'display_name': 'Molecular Biology'}, 'field': {'id': 'https://openalex.org/fields/13', 'display_name': 'Biochemistry, Genetics and Molecular Biology'}, 'domain': {'id': 'https://openalex.org/domains/1', 'display_name': 'Life Sciences'}}, {'id': 'https://openalex.org/T10604', 'display_name': 'RNA Research and Splicing', 'score': 0.9889, 'subfield': {'id': 'https://openalex.org/subfields/1312', 'display_name': 'Molecular Biology'}, 'field': {'id': 'https://openalex.org/fields/13', 'display_name': 'Biochemistry, Genetics and Molecular Biology'}, 'domain': {'id': 'https://openalex.org/domains/1', 'display_name': 'Life Sciences'}}], 'keywords': [{'id': 'https://openalex.org/keywords/sarcolemma', 'display_name': 'Sarcolemma', 'score': 0.7125126}, {'id': 'https://openalex.org/keywords/utrophin', 'display_name': 'Utrophin', 'score': 0.68175614}, {'id': 'https://openalex.org/keywords/exon-skipping', 'display_name': 'Exon skipping', 'score': 0.495921}, {'id': 'https://openalex.org/keywords/dysferlin', 'display_name': 'Dysferlin', 'score': 0.47861952}], 'concepts': [{'id': 'https://openalex.org/C2780394045', 'wikidata': 'https://www.wikidata.org/wiki/Q412285', 'display_name': 'Dystrophin', 'level': 3, 'score': 0.9079968}, {'id': 'https://openalex.org/C2778943923', 'wikidata': 'https://www.wikidata.org/wiki/Q1648484', 'display_name': 'Duchenne muscular dystrophy', 'level': 2, 'score': 0.8313564}, {'id': 'https://openalex.org/C199096232', 'wikidata': 'https://www.wikidata.org/wiki/Q1545001', 'display_name': 'Sarcolemma', 'level': 3, 'score': 0.7125126}, {'id': 'https://openalex.org/C78343239', 'wikidata': 'https://www.wikidata.org/wiki/Q22683570', 'display_name': 'Utrophin', 'level': 4, 'score': 0.68175614}, {'id': 'https://openalex.org/C2779030066', 'wikidata': 'https://www.wikidata.org/wiki/Q1137767', 'display_name': 'Muscular dystrophy', 'level': 2, 'score': 0.6644765}, {'id': 'https://openalex.org/C2778776201', 'wikidata': 'https://www.wikidata.org/wiki/Q3474283', 'display_name': 'Exon skipping', 'level': 5, 'score': 0.495921}, {'id': 'https://openalex.org/C37113945', 'wikidata': 'https://www.wikidata.org/wiki/Q14885337', 'display_name': 'Dysferlin', 'level': 3, 'score': 0.47861952}, {'id': 'https://openalex.org/C71924100', 'wikidata': 'https://www.wikidata.org/wiki/Q11190', 'display_name': 'Medicine', 'level': 0, 'score': 0.46728975}, {'id': 'https://openalex.org/C2777300911', 'wikidata': 'https://www.wikidata.org/wiki/Q692536', 'display_name': 'Myopathy', 'level': 2, 'score': 0.42682222}, {'id': 'https://openalex.org/C86803240', 'wikidata': 'https://www.wikidata.org/wiki/Q420', 'display_name': 'Biology', 'level': 0, 'score': 0.39161748}, {'id': 'https://openalex.org/C54355233', 'wikidata': 'https://www.wikidata.org/wiki/Q7162', 'display_name': 'Genetics', 'level': 1, 'score': 0.38374007}, {'id': 'https://openalex.org/C60644358', 'wikidata': 'https://www.wikidata.org/wiki/Q128570', 'display_name': 'Bioinformatics', 'level': 1, 'score': 0.3707192}, {'id': 'https://openalex.org/C126322002', 'wikidata': 'https://www.wikidata.org/wiki/Q11180', 'display_name': 'Internal medicine', 'level': 1, 'score': 0.3030941}, {'id': 'https://openalex.org/C36823959', 'wikidata': 'https://www.wikidata.org/wiki/Q373027', 'display_name': 'Exon', 'level': 3, 'score': 0.2905071}, {'id': 'https://openalex.org/C104317684', 'wikidata': 'https://www.wikidata.org/wiki/Q7187', 'display_name': 'Gene', 'level': 2, 'score': 0.25450534}, {'id': 'https://openalex.org/C2779959927', 'wikidata': 'https://www.wikidata.org/wiki/Q1048687', 'display_name': 'Skeletal muscle', 'level': 2, 'score': 0.24203447}, {'id': 'https://openalex.org/C194583182', 'wikidata': 'https://www.wikidata.org/wiki/Q21114084', 'display_name': 'Alternative splicing', 'level': 4, 'score': 0.06530577}], 'mesh': [{'descriptor_ui': 'D017277', 'descriptor_name': 'Drug Approval', 'qualifier_ui': '', 'qualifier_name': None, 'is_major_topic': True}, {'descriptor_ui': 'D060172', 'descriptor_name': 'Morpholinos', 'qualifier_ui': 'Q000627', 'qualifier_name': 'therapeutic use', 'is_major_topic': True}, {'descriptor_ui': 'D020388', 'descriptor_name': 'Muscular Dystrophy, Duchenne', 'qualifier_ui': 'Q000188', 'qualifier_name': 'drug therapy', 'is_major_topic': True}, {'descriptor_ui': 'D006801', 'descriptor_name': 'Humans', 'qualifier_ui': '', 'qualifier_name': None, 'is_major_topic': False}, {'descriptor_ui': 'D008297', 'descriptor_name': 'Male', 'qualifier_ui': '', 'qualifier_name': None, 'is_major_topic': False}, {'descriptor_ui': 'D060172', 'descriptor_name': 'Morpholinos', 'qualifier_ui': '', 'qualifier_name': None, 'is_major_topic': False}, {'descriptor_ui': 'D020388', 'descriptor_name': 'Muscular Dystrophy, Duchenne', 'qualifier_ui': '', 'qualifier_name': None, 'is_major_topic': False}, {'descriptor_ui': 'D009841', 'descriptor_name': 'Oligonucleotides', 'qualifier_ui': '', 'qualifier_name': None, 'is_major_topic': False}, {'descriptor_ui': 'D014481', 'descriptor_name': 'United States', 'qualifier_ui': '', 'qualifier_name': None, 'is_major_topic': False}, {'descriptor_ui': 'D014486', 'descriptor_name': 'United States Food and Drug Administration', 'qualifier_ui': '', 'qualifier_name': None, 'is_major_topic': False}], 'locations_count': 4, 'locations': [{'is_oa': True, 'landing_page_url': 'https://doi.org/10.1038/mt.2016.188', 'pdf_url': 'https://www.cell.com/article/S1525001616454390/pdf', 'source': {'id': 'https://openalex.org/S51596929', 'display_name': 'Molecular Therapy', 'issn_l': '1525-0016', 'issn': ['1525-0016', '1525-0024'], 'is_oa': False, 'is_in_doaj': False, 'is_core': True, 'host_organization': 'https://openalex.org/P4310320990', 'host_organization_name': 'Elsevier BV', 'host_organization_lineage': ['https://openalex.org/P4310320990'], 'host_organization_lineage_names': ['Elsevier BV'], 'type': 'journal'}, 'license': 'cc-by-nc-nd', 'license_id': 'https://openalex.org/licenses/cc-by-nc-nd', 'version': 'publishedVersion', 'is_accepted': True, 'is_published': True}, {'is_oa': True, 'landing_page_url': 'https://europepmc.org/articles/pmc5154484', 'pdf_url': 'https://europepmc.org/articles/pmc5154484?pdf=render', 'source': {'id': 'https://openalex.org/S4306400806', 'display_name': 'Europe PMC (PubMed Central)', 'issn_l': None, 'issn': None, 'is_oa': True, 'is_in_doaj': False, 'is_core': False, 'host_organization': 'https://openalex.org/I1303153112', 'host_organization_name': 'European Bioinformatics Institute', 'host_organization_lineage': ['https://openalex.org/I1303153112'], 'host_organization_lineage_names': ['European Bioinformatics Institute'], 'type': 'repository'}, 'license': 'cc-by-nc-nd', 'license_id': 'https://openalex.org/licenses/cc-by-nc-nd', 'version': 'publishedVersion', 'is_accepted': True, 'is_published': True}, {'is_oa': True, 'landing_page_url': 'https://www.ncbi.nlm.nih.gov/pmc/articles/PMC5154484', 'pdf_url': None, 'source': {'id': 'https://openalex.org/S2764455111', 'display_name': 'PubMed Central', 'issn_l': None, 'issn': None, 'is_oa': True, 'is_in_doaj': False, 'is_core': False, 'host_organization': 'https://openalex.org/I1299303238', 'host_organization_name': 'National Institutes of Health', 'host_organization_lineage': ['https://openalex.org/I1299303238'], 'host_organization_lineage_names': ['National Institutes of Health'], 'type': 'repository'}, 'license': None, 'license_id': None, 'version': 'publishedVersion', 'is_accepted': True, 'is_published': True}, {'is_oa': False, 'landing_page_url': 'https://pubmed.ncbi.nlm.nih.gov/27916994', 'pdf_url': None, 'source': {'id': 'https://openalex.org/S4306525036', 'display_name': 'PubMed', 'issn_l': None, 'issn': None, 'is_oa': False, 'is_in_doaj': False, 'is_core': False, 'host_organization': 'https://openalex.org/I1299303238', 'host_organization_name': 'National Institutes of Health', 'host_organization_lineage': ['https://openalex.org/I1299303238'], 'host_organization_lineage_names': ['National Institutes of Health'], 'type': 'repository'}, 'license': None, 'license_id': None, 'version': None, 'is_accepted': False, 'is_published': False}], 'best_oa_location': {'is_oa': True, 'landing_page_url': 'https://doi.org/10.1038/mt.2016.188', 'pdf_url': 'https://www.cell.com/article/S1525001616454390/pdf', 'source': {'id': 'https://openalex.org/S51596929', 'display_name': 'Molecular Therapy', 'issn_l': '1525-0016', 'issn': ['1525-0016', '1525-0024'], 'is_oa': False, 'is_in_doaj': False, 'is_core': True, 'host_organization': 'https://openalex.org/P4310320990', 'host_organization_name': 'Elsevier BV', 'host_organization_lineage': ['https://openalex.org/P4310320990'], 'host_organization_lineage_names': ['Elsevier BV'], 'type': 'journal'}, 'license': 'cc-by-nc-nd', 'license_id': 'https://openalex.org/licenses/cc-by-nc-nd', 'version': 'publishedVersion', 'is_accepted': True, 'is_published': True}, 'sustainable_development_goals': [{'id': 'https://metadata.un.org/sdg/3', 'display_name': 'Good health and well-being', 'score': 0.74}], 'grants': [], 'datasets': [], 'versions': [], 'referenced_works_count': 7, 'referenced_works': ['https://openalex.org/W1533447420', 'https://openalex.org/W1976354092', 'https://openalex.org/W2063512628', 'https://openalex.org/W2157085976', 'https://openalex.org/W2158156749', 'https://openalex.org/W2234725241', 'https://openalex.org/W2518024229'], 'related_works': ['https://openalex.org/W2755970341', 'https://openalex.org/W2474924864', 'https://openalex.org/W2351942144', 'https://openalex.org/W2189501906', 'https://openalex.org/W2142169677', 'https://openalex.org/W2102878417', 'https://openalex.org/W2027064784', 'https://openalex.org/W2020769270', 'https://openalex.org/W2006833948', 'https://openalex.org/W2003601635'], 'abstract_inverted_index': {'Eeplirsen': [0], '(Sarepta': [1], 'Inc.,': [2], 'Boston,': [3], 'MA)': [4], 'is': [5, 63, 193, 539, 1095, 1112, 1421], 'a': [6, 216, 460, 545, 765, 782, 906, 930, 944, 962, 967, 988, 1048, 1183, 1189, 1246, 1253, 1310, 1315, 1345, 1350, 1365, 1385], '30-mer': [7], 'phosphomorpholidate': [8], 'oligonucleotide': [9], 'that': [10, 43, 71, 94, 214, 687, 797, 813, 951, 1008, 1094, 1110, 1213, 1248, 1422], 'has': [11, 688, 1121], 'been': [12, 689, 1056, 1122, 1272], 'evaluated': [13], 'in': [14, 32, 67, 114, 182, 186, 233, 264, 306, 320, 332, 357, 378, 429, 456, 541, 591, 594, 620, 646, 659, 815, 828, 839, 1029, 1032, 1052, 1149, 1359, 1414], 'young': [15], 'male': [16, 36], 'patients': [17, 265, 330, 395, 414, 661, 724, 746, 755, 774, 850, 1053, 1085, 1395], 'with': [18, 218, 242, 266, 415, 451, 662, 666, 854, 1299, 1363], 'the': [19, 48, 68, 73, 99, 115, 123, 168, 196, 222, 226, 327, 388, 391, 397, 417, 452, 483, 551, 608, 671, 676, 681, 798, 802, 816, 829, 840, 848, 857, 875, 901, 921, 980, 982, 1005, 1043, 1068, 1074, 1081, 1108, 1144, 1171, 1179, 1193, 1197, 1210, 1243, 1284, 1295, 1319, 1324, 1329, 1335, 1354, 1373, 1380, 1383, 1398, 1416], 'devastating': [20], 'disease': [21], 'known': [22], 'as': [23, 136, 302, 423, 567, 585, 702, 1086, 1120, 1187, 1192, 1338], 'Duchenne': [24, 51, 102, 140, 267, 358, 457, 571, 647, 706, 1132], 'muscular': [25, 52, 103, 141, 171, 268, 359, 458, 543, 572, 648, 707, 1311, 1346], 'dystrophy': [26, 269, 1312, 1347], '(DMD).': [27], 'This': [28, 526, 1156], 'X-linked': [29], 'disease,': [30], 'found': [31, 428, 540, 1148], 'approximately': [33], '1/3,500–1/5,000': [34], 'live': [35], 'births,1Robinson-Hamm': [37], 'J': [38, 89, 129, 154, 336, 338, 346, 560, 625, 627, 635, 695, 1003], 'Gersbach': [39, 90], 'C': [40, 91, 444], 'Gene': [41, 92, 516], 'therapies': [42, 93], 'restore': [44, 95], 'dystrophin': [45, 69, 96, 116, 191, 231, 262, 449, 534, 601, 1030, 1101], 'expression': [46, 97, 450], 'for': [47, 98, 139, 167, 380, 512, 570, 705, 742, 764, 790, 896, 900, 918, 1047, 1163, 1250, 1264], 'treatment': [49, 100, 169, 881], 'of': [50, 80, 101, 125, 170, 230, 237, 350, 390, 393, 413, 448, 486, 491, 550, 586, 600, 611, 639, 767, 784, 792, 847, 856, 865, 874, 932, 938, 973, 1026, 1088, 1143, 1146, 1245, 1283, 1326, 1331, 1376, 1400], 'dystrophy.Hum': [53, 104], 'Genet.': [54, 105], '2016;': [55, 106, 362, 651, 1016], '135:': [56, 107], '1029-1040Crossref': [57, 108], 'PubMed': [58, 109, 177, 290, 365, 475, 521, 654], 'Scopus': [59, 110, 150, 178, 291, 366, 476, 522, 581, 655, 716], '(50)': [60, 111], 'Google': [61, 112, 152, 180, 293, 368, 478, 524, 583, 657, 718], 'Scholar': [62, 113, 181, 294, 369, 584, 719, 1018, 1140, 1208, 1322, 1357], 'characterized': [64], 'by': [65, 311, 326, 401, 406, 616, 691, 943, 1059, 1063, 1378], 'mutations': [66, 83], 'gene': [70], 'take': [72], 'messenger': [74], 'RNA': [75], '(mRNA)': [76], 'reading': [77], 'frame': [78], 'out': [79], 'frame.': [81], 'These': [82], 'can': [84, 372, 426, 1381], 'produce': [85, 1389, 1413], 'premature': [86], 'stop': [87], 'codons1Robinson-Hamm': [88], 'mRNA,': [117], 'leading': [118, 1254], 'to': [119, 205, 210, 225, 309, 482, 537, 554, 727, 778, 825, 833, 836, 870, 880, 927, 960, 979, 995, 1037, 1072, 1080, 1106, 1130, 1228, 1241, 1260, 1274, 1323, 1361], 'mRNA': [120], 'degradation': [121], 'and': [122, 207, 235, 239, 245, 261, 324, 509, 593, 723, 872, 899, 910, 1067, 1257, 1292, 1328], 'loss': [124, 229], 'protein': [126, 192, 213, 602, 1147], 'synthesis2Van': [127], 'Deutekom': [128, 556, 559, 694], 'Kimpe': [130, 561, 696], 'S': [131, 247, 255, 440, 562, 697], 'Campion': [132, 563, 698], 'G': [133, 564, 699], 'Antisense': [134, 565, 700], 'oligonucleotides': [135, 566, 701], 'personalized': [137, 568, 703], 'medicine': [138, 569, 704], 'dystrophy.Drug': [142, 573, 708], 'Discov': [143, 574, 709], 'Today': [144, 575, 710], 'Ther': [145, 576, 711], 'Strateg.': [146, 577, 712], '2013;': [147, 174, 578, 713], '10:': [148, 579, 714], 'e149-e156Crossref': [149, 580, 715], '(7)': [151, 582, 717], 'Scholar,3Mendell': [153], 'Rodino-Klapac': [155], 'L': [156, 162, 164, 253, 340, 342, 438, 629, 631], 'Sahenk': [157], 'Z': [158], 'Roush': [159], 'K': [160, 257, 344, 506, 633], 'Bird': [161], 'Lowes': [163, 339, 628], 'et': [165, 258, 347, 445, 507, 557, 618, 636], 'al.Eteplirsen': [166], 'dystrophy.Ann': [172, 360, 649], 'Neurol.': [173, 361, 466, 650], '74:': [175], '637-647Crossref': [176], '(538)': [179], 'striated': [183], 'muscle,': [184, 238], 'resulting': [185, 319], 'catastrophic': [187], 'clinical': [188, 1049], 'consequences.': [189], 'The': [190, 228, 794], 'located': [194], 'beneath': [195], 'sarcolemma': [197], '(the': [198], 'muscle': [199, 315, 1034, 1151], 'cell': [200], 'membrane).': [201], 'Through': [202], 'N-terminal': [203], 'binding': [204, 209], 'F-actin': [206], 'C-terminal': [208], 'β-dystroglycan': [211], '(a': [212], 'forms': [215], 'complex': [217], 'dystrophin),': [219], 'it': [220, 924, 1009, 1117, 1214, 1423], 'connects': [221], 'cellular': [223], 'cytoskeleton': [224], 'sarcolemma.': [227], 'results': [232, 795, 1079], 'inflammation': [234], 'degeneration': [236], 'its': [240, 1402], 'replacement': [241], 'fibrotic': [243], 'tissue': [244, 1035], 'fat.4Cirak': [246], 'Arechavala-Gomeza': [248, 435], 'V': [249, 436], 'Guglieri': [250], 'M': [251, 1308, 1343], 'Feng': [252, 437], 'Torelli': [254], 'Anthony': [256], 'al.Exon': [259], 'skipping': [260, 490, 514], 'restoration': [263, 447, 599], 'after': [270], 'systemic': [271], 'phosphorodiamidate': [272], 'morpholino': [273, 453], 'oligomer': [274, 454], 'treatment:': [275], 'an': [276, 420, 528, 604, 750, 1027, 1089, 1160, 1164], 'open-label,': [277], 'phase': [278], '2,': [279], 'dose-escalation': [280], 'study.Lancet.': [281], '2011;': [282], '378:': [283], '595-605Abstract': [284], 'Full': [285, 287, 470, 472], 'Text': [286, 288, 471, 473], 'PDF': [289, 474], '(699)': [292], 'Sufferers': [295], 'from': [296, 375, 822, 1083], 'DMD': [297, 329, 394, 663], 'progressively': [298], 'lose': [299], 'neuromuscular': [300], 'function': [301], 'they': [303, 1238], 'age,': [304], 'culminating': [305], 'their': [307, 312, 333, 897, 1060, 1229, 1265], 'inability': [308], 'walk': [310, 678], 'mid-teens.': [313], 'Sadly,': [314], 'deterioration': [316], 'continues': [317], 'unabated,': [318], 'cardiomyopathy,': [321], 'respiratory': [322], 'failure,': [323], 'death': [325], 'time': [328], 'are': [331, 404], 'third': [334], 'decade.5Mendell': [335], 'Goemans': [337, 499, 626], 'Alfano': [341, 630], 'Berry': [343, 632], 'Shao': [345, 634], 'al.Longitudinal': [348, 637], 'effect': [349, 638], 'eteplirsen': [351, 640, 735, 933, 998, 1111], 'versus': [352, 641], 'historical': [353, 642, 786, 817, 841, 858, 974], 'control': [354, 643, 859], 'on': [355, 644, 749, 801, 965, 1013, 1128, 1391], 'ambulation': [356, 645], '79:': [363, 652], '257-271Crossref': [364, 653], '(366)': [367, 656], 'As': [370, 1167], 'I': [371], 'assure': [373], 'you': [374], 'personal': [376], 'experience': [377], 'caring': [379], 'such': [381, 966, 1364], 'patients,': [382, 831, 895, 1267], 'words': [383], 'cannot': [384], 'adequately': [385], 'express': [386], 'either': [387], 'heartbreak': [389], 'parents': [392], 'or': [396, 738, 761, 883, 1396], 'suffering': [398], 'patiently': [399], 'endured': [400], 'those': [402], 'who': [403, 903, 1054, 1333], 'afflicted': [405], 'this': [407, 669, 889], 'terrible': [408], 'ailment.': [409], 'In': [410, 668, 920, 976, 1182], 'roughly': [411], '13%': [412], 'DMD,': [416], 'molecular': [418], 'lesion,': [419], 'out-of-frame': [421], 'deletion': [422], 'mentioned': [424], 'above,': [425], 'be': [427, 993, 1038, 1104, 1154, 1159], 'exons': [430], '47–63': [431], '(ref.': [432, 494, 623], '6Kinali': [433], 'A': [434, 496, 498, 502, 1124], 'Cirak': [439], 'Hunt': [441], 'D': [442, 504], 'Adkin': [443], 'al.Local': [446], 'AVI-4658': [455], 'dystrophy:': [459], 'single-blind,': [461], 'placebo-controlled': [462, 990, 1092], 'dose-escalation,': [463], 'proof-of-concept': [464], 'study.Lancet': [465], '2009;': [467], '8:': [468], '918-928Abstract': [469], '(572)': [477], 'Scholar).': [479, 525], 'Eteplirsen,': [480, 1301], 'targeted': [481], 'splice-donor': [484], 'region': [485], 'exon': [487, 492, 513], '51,': [488, 1305], 'induces': [489], '51': [493, 1411], '7Aartsma-Rus': [495], 'Ferline': [497], 'N': [500], 'Pasmooij': [501], 'Wells': [503], 'Bushby': [505], 'al.Translational': [508], 'regulatory': [510, 692], 'challenges': [511], 'therapies.Hum': [515], 'Ther.': [517], '2014;': [518], '25:': [519], '885-892Crossref': [520], '(38)': [523], 'yields': [527], 'in-frame,': [529], 'truncated,': [530], 'yet': [531, 1012], 'partially': [532], 'functional': [533, 673], 'protein,': [535], 'similar': [536], 'what': [538, 1114], 'Becker': [542], 'dystrophy,': [544], 'much': [546, 1025, 1100], 'less': [547, 811, 1041], 'severe': [548], 'form': [549], 'disease.': [552], 'According': [553], 'van': [555], 'al.,2Van': [558], '2013': [587], 'more': [588, 1392], 'than': [589, 812, 1042, 1221, 1393], '100': [590, 837, 936], 'vitro': [592], 'vivo': [595], 'studies': [596, 613], 'had': [597, 775, 953, 999, 1021, 1055, 1070], 'demonstrated': [598, 796, 1024], 'via': [603], 'exon-skipping': [605], 'strategy.': [606], 'Perhaps': [607], 'most': [609], 'critical': [610], 'these': [612], 'was': [614, 675, 808, 925, 985, 1175, 1233, 1306], 'published': [615], 'Mendell': [617], 'al.': [619], 'early': [621], '2016': [622, 1065, 1205], '5Mendell': [624], 'Scholar),': [658], 'which': [660], 'were': [664, 721, 788, 862], 'treated': [665, 1084], 'eteplirsen.': [667], 'study,': [670], 'primary': [672, 683], 'assessment': [674], 'six-minute': [677], 'test': [679, 804], '(6MWT),': [680], '"only': [682], 'validated': [684], 'efficacy': [685], 'endpoint': [686], 'accepted': [690], 'authorities."2Van': [693], 'Treatments': [720], 'double-blinded': [722], 'randomly': [725], 'assigned': [726], 'three': [728, 769, 844], 'cohorts': [729], '(n': [730, 970], '=': [731, 971], '4': [732], 'each):': [733], 'placebo;': [734], '30': [736], 'mg/kg': [737, 740], '50': [739, 762], 'weekly': [741], '24': [743], 'weeks.': [744], 'Subsequently,': [745], 'received': [747, 757], 'drug': [748, 759, 780, 1133, 1247, 1313, 1348], 'open-label': [751, 758], 'basis,': [752], 'while': [753], 'placebo': [754, 773], 'also': [756], '(30': [760], 'mg/kg)': [763], 'span': [766], 'over': [768, 777, 935], 'years.': [770], 'Because': [771], 'all': [772], 'crossed': [776], 'active': [779], 'treatment,': [781, 871], 'group': [783], 'pooled': [785], 'controls': [787, 818], 'employed': [789], 'purposes': [791], 'comparison.': [793], 'distance': [799], 'lost': [800, 814, 851], '6MWT': [803], 'at': [805, 1318, 1353], '36': [806], 'months': [807], '151': [809], 'm': [810, 827, 838], '(P': [819], '<': [820, 1206], '0.01;': [821], 'about': [823, 834, 1415], '350': [824, 835], '250': [826], 'eteplirsen-treated': [830, 849], 'compared': [832, 853], 'controls).': [842], 'After': [843], 'years,': [845], '16.7%': [846], 'ambulation,': [852], '46.2%': [855], 'patients.': [860, 1098], 'There': [861, 1232], 'no': [863], 'reports': [864], 'serious': [866], 'adverse': [867, 877], 'advents': [868], 'related': [869], 'none': [873], 'observed': [876], 'events': [878], 'led': [879], 'interruptions': [882], 'dose': [884], 'adjustments.': [885], 'You': [886], 'might': [887], 'think': [888], 'story': [890], 'would': [891, 992, 1215], 'quickly': [892], 'end': [893], 'happily—for': [894], 'parents,': [898], 'doctors': [902], 'treat': [904], 'DMD—as': [905], 'joyful': [907], 'US': [908], 'Food': [909], 'Drug': [911, 1286], 'Administration': [912], '(FDA)': [913], 'overwhelmingly': [914], 'approved': [915], "Sarepta's": [916, 1014], 'application': [917], 'marketing.': [919], 'short': [922], 'term,': [923], 'not': [926, 954, 959, 1158, 1176, 1234], 'be.': [928], 'Despite': [929], '"legion"': [931], 'advocates—including': [934], 'members': [937], 'Congress—an': [939], 'FDA': [940, 957, 1006, 1069, 1082, 1109, 1126, 1225, 1296, 1404], 'advisory': [941], 'panel,': [942], '6–7': [945], 'vote,': [946], "wasn't": [947], 'convinced.': [948], 'It': [949], 'seems': [950], 'Sarepta': [952, 1020, 1066, 1115, 1387], 'heeded': [955], 'repeated': [956], 'advice': [958], 'perform': [961], 'trial': [963, 991, 1093], 'based': [964], 'small': [968], 'number': [969], '11)': [972], 'controls.': [975], 'fact,': [977], 'according': [978], 'FDA,': [981], 'entire': [983], 'study': [984], 'too': [986], 'small;': [987], 'larger,': [989], 'necessary': [994, 1046], 'determine': [996], 'if': [997, 1409], 'truly': [1000], 'helped': [1001, 1058], 'anyone.8Carroll': [1002], 'Updated:': [1004], 'decides': [1007], "can't": [1010], 'decide': [1011], 'eteplirsen.FierceBiotech.': [1015], '25<http://www.fiercebiotech.com/fda-decides-it-can-t-decide-yet-sarepta-s-eteplirsen>Google': [1017], 'Moreover,': [1019], 'apparently': [1022], 'never': [1023], 'increase': [1028], 'production': [1031, 1102], 'affected': [1033], '(0.28%,': [1036], 'precise,': [1039], 'far': [1040], '10%': [1044], 'deemed': [1045], 'impact),': [1050], 'even': [1051, 1341], 'clinically': [1057], 'drug.': [1061], 'Fortunately,': [1062], 'June': [1064, 1137], 'come': [1071], 'terms;': [1073], 'company': [1075], 'will': [1076, 1103, 1153, 1412], 'submit': [1077], 'biopsy': [1078], 'part': [1087], 'additional,': [1090], 'larger': [1091], 'currently': [1096], 'accruing': [1097], 'How': [1099], 'sufficient': [1105, 1235], 'convince': [1107], 'doing': [1113], 'claims': [1116], 'does?': [1118], 'Perhaps,': [1119], 'suggested,9Feuerstein': [1123], 'Sarepta,': [1125], 'compromise': [1127], 'plan': [1129], 'secure': [1131], 'approval': [1134], 'TheStreet': [1135], '7': [1136], '2016.https://www.thestreet.com/story/13598536/1/sarepta-fda-compromise-on-plan-to-secure-duchenne-drug-approval.htmlDate:': [1138], '2016Google': [1139, 1321, 1356], 'only': [1141], '0.9%': [1142], 'amount': [1145], 'normal': [1150], 'cells': [1152], 'necessary.': [1155], 'should': [1157, 1407], 'insurmountable': [1161], 'barrier': [1162], '"active"': [1165], 'oligonucleotide.': [1166], 'one': [1168], 'could': [1169], 'imagine,': [1170], "FDA's": [1172, 1285, 1336], 'initial': [1173], 'position': [1174], 'popular': [1177], 'among': [1178], 'advocacy': [1180], 'set.': [1181], 'recent': [1184], 'opinion': [1185], 'piece,': [1186], 'learned': [1188], 'media': [1190], 'organ': [1191], 'Wall': [1194, 1200], 'Street': [1195, 1201], "Journal10Where's": [1196], 'Drug,': [1198], 'FDA?': [1199], 'Journal': [1202], '5': [1203], 'July': [1204], 'http://www.wsj.com/articles/wheres-the-drug-fda-1467413266>.Google': [1207], 'berated': [1209], 'agency,': [1211], 'saying': [1212], 'rather': [1216], '"check': [1217], 'off': [1218], 'procedural': [1219], 'boxes': [1220], 'confront…realities."': [1222], 'Nevertheless,': [1223], 'numerous': [1224], 'scientists': [1226], 'adhered': [1227], 'original': [1230], 'analysis.': [1231], 'scientific': [1236], 'information,': [1237], 'emphatically': [1239], 'stated,': [1240], 'justify': [1242], 'marketing': [1244], 'retailed': [1249], '$300,000/patient/year.': [1251], '(Anthem,': [1252], 'carrier,': [1255], 'agreed': [1256, 1298], 'eventually': [1258], 'refused': [1259], 'cover': [1261], "eteplirsen's": [1262], 'cost': [1263], 'insured': [1266], 'although': [1268], 'other': [1269], 'carriers': [1270], 'have': [1271], 'willing': [1273], 'approve': [1275], 'coverage.)': [1276], 'On': [1277], '19': [1278], 'September,': [1279], 'Janet': [1280], 'Woodcock,': [1281], 'Director': [1282], 'Division,': [1287], 'overruled': [1288], 'her': [1289], 'own': [1290], 'scientists,': [1291], 'Robert': [1293], 'Califf,': [1294], 'commissioner,': [1297], 'her.': [1300], 'now': [1302], 'renamed': [1303], 'Exondys': [1304, 1410], 'approved,11Herper': [1307], 'Approving': [1309, 1344], 'ignites': [1314, 1349], 'civil': [1316, 1351], 'war': [1317, 1352], 'FDA.http://www.forbes.com/sites/matthewherper/2016/09/20/approving-a-muscular-dystrophy-drug-ignites-civil-war-at-the-fda/#75ee1264353dDate:': [1320, 1355], 'delight': [1325], 'many': [1327], 'fury': [1330], 'some,': [1332], 'lamented': [1334], 'decision': [1337], '"this': [1339], "isn't": [1340], 'science."11Herper': [1342], 'But': [1358], 'having': [1360], 'deal': [1362], 'politically': [1366], 'charged': [1367], 'question,': [1368], 'perhaps': [1369], 'Dr.': [1370], 'Woodcock': [1371], 'took': [1372], 'optimal': [1374], 'course': [1375], 'action': [1377], 'kicking': [1379], 'down': [1382], 'road': [1384], 'bit;': [1386], 'must': [1388], 'data': [1390], '12': [1394], 'run': [1397], 'risk': [1399], 'losing': [1401], 'hard-won': [1403], 'approval.': [1405], 'We': [1406], 'know': [1408], 'year': [1417], '2020.': [1418], 'My': [1419], 'belief': [1420], 'ultimately': [1424], 'will.': [1425]}, 'cited_by_api_url': 'https://api.openalex.org/works?filter=cites:W2560408974', 'counts_by_year': [{'year': 2024, 'cited_by_count': 7}, {'year': 2023, 'cited_by_count': 4}, {'year': 2022, 'cited_by_count': 3}, {'year': 2021, 'cited_by_count': 10}, {'year': 2020, 'cited_by_count': 5}, {'year': 2019, 'cited_by_count': 10}, {'year': 2018, 'cited_by_count': 13}, {'year': 2017, 'cited_by_count': 12}], 'updated_date': '2024-12-27T20:22:54.517813', 'created_date': '2016-12-16'}