{"id":"https://openalex.org/W4405749685","doi":"https://doi.org/10.1016/j.jval.2024.10.3462","title":"PCR220 Patient-Reported Outcomes in LGMD 2E/R4, 2D/R3 and 2C/R5: Descriptive Analysis of Baseline PROMIS Data From the JOURNEY Natural History Study","display_name":"PCR220 Patient-Reported Outcomes in LGMD 2E/R4, 2D/R3 and 2C/R5: Descriptive Analysis of Baseline PROMIS Data From the JOURNEY Natural History Study","publication_year":2024,"publication_date":"2024-12-01","ids":{"openalex":"https://openalex.org/W4405749685","doi":"https://doi.org/10.1016/j.jval.2024.10.3462"},"language":"en","primary_location":{"id":"doi:10.1016/j.jval.2024.10.3462","doi":"https://doi.org/10.1016/j.jval.2024.10.3462","is_oa":true,"landing_page_url":"https://doi.org/10.1016/j.jval.2024.10.3462","pdf_url":"https://www.valueinhealthjournal.com/article/S1098-3015(24)06347-2/pdf","source":{"id":"https://openalex.org/S678965","display_name":"Value in Health","issn_l":"1098-3015","issn":["1098-3015","1524-4733"],"is_oa":false,"is_in_doaj":false,"is_core":true,"listed_in":["abdc-a","anvur-class-a","anvur-scientific","cwts-core","dongbi-a","doyens","jufo-2","ki-jl-2","medline","norway-2","poland-140","russia-white-list-1","vabb-shw"],"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":null,"license_id":null,"version":"publishedVersion","is_accepted":true,"is_published":true,"raw_source_name":"Value in Health","raw_type":"journal-article"},"volume":null,"type":"conference-abstract","is_traditional":false,"indexed_in":["crossref"],"open_access":{"is_oa":true,"oa_status":"bronze","oa_url":"https://www.valueinhealthjournal.com/article/S1098-3015(24)06347-2/pdf","any_repository_has_fulltext":false},"authorships":[{"author_position":"first","author":{"id":"https://openalex.org/A5114177982","display_name":"I. Filipovic Audhya","orcid":null,"observed_orcids":[]},"institutions":[{"id":"https://openalex.org/I4210148120","display_name":"Sarepta Therapeutics (United States)","ror":"https://ror.org/054f2wp19","country_code":"US","type":"company","lineage":["https://openalex.org/I4210148120"]}],"countries":["US"],"is_corresponding":false,"raw_author_name":"I. Filipovic Audhya","raw_affiliation_strings":["Sarepta Therapeutics, Inc., Cambridge, MA, USA"],"raw_orcid":null,"affiliations":[{"raw_affiliation_string":"Sarepta Therapeutics, Inc., Cambridge, MA, USA","institution_ids":["https://openalex.org/I4210148120"]}]},{"author_position":"middle","author":{"id":"https://openalex.org/A5089880100","display_name":"Alise Nacson","orcid":"https://orcid.org/0009-0004-9532-4274","observed_orcids":["https://orcid.org/0009-0004-9532-4274"]},"institutions":[{"id":"https://openalex.org/I4210148120","display_name":"Sarepta Therapeutics (United States)","ror":"https://ror.org/054f2wp19","country_code":"US","type":"company","lineage":["https://openalex.org/I4210148120"]}],"countries":["US"],"is_corresponding":false,"raw_author_name":"A. Nacson","raw_affiliation_strings":["Sarepta Therapeutics, Inc., Cambridge, MA, USA"],"raw_orcid":null,"affiliations":[{"raw_affiliation_string":"Sarepta Therapeutics, Inc., Cambridge, MA, USA","institution_ids":["https://openalex.org/I4210148120"]}]},{"author_position":"middle","author":{"id":null,"display_name":"P. Fromy","orcid":null,"observed_orcids":[]},"institutions":[],"countries":[],"is_corresponding":false,"raw_author_name":"P. Fromy","raw_affiliation_strings":["SeeingTheta, Samur, Saumur, France"],"raw_orcid":null,"affiliations":[{"raw_affiliation_string":"SeeingTheta, Samur, Saumur, France","institution_ids":[]}]},{"author_position":"middle","author":{"id":"https://openalex.org/A5029849735","display_name":"Katherine L. Gooch","orcid":"https://orcid.org/0009-0000-6382-614X","observed_orcids":["https://orcid.org/0009-0000-6382-614X"]},"institutions":[{"id":"https://openalex.org/I4210148120","display_name":"Sarepta Therapeutics (United States)","ror":"https://ror.org/054f2wp19","country_code":"US","type":"company","lineage":["https://openalex.org/I4210148120"]}],"countries":["US"],"is_corresponding":false,"raw_author_name":"K.L. Gooch","raw_affiliation_strings":["Sarepta Therapeutics, Inc., Cambridge, MA, USA"],"raw_orcid":null,"affiliations":[{"raw_affiliation_string":"Sarepta Therapeutics, Inc., Cambridge, MA, USA","institution_ids":["https://openalex.org/I4210148120"]}]},{"author_position":"last","author":{"id":"https://openalex.org/A5025427506","display_name":"H. Stevenson","orcid":null,"observed_orcids":[]},"institutions":[{"id":"https://openalex.org/I4210148120","display_name":"Sarepta Therapeutics (United States)","ror":"https://ror.org/054f2wp19","country_code":"US","type":"company","lineage":["https://openalex.org/I4210148120"]}],"countries":["US"],"is_corresponding":false,"raw_author_name":"H. Stevenson","raw_affiliation_strings":["Sarepta Therapeutics, Inc., Cambridge, MA, USA"],"raw_orcid":null,"affiliations":[{"raw_affiliation_string":"Sarepta Therapeutics, Inc., Cambridge, MA, USA","institution_ids":["https://openalex.org/I4210148120"]}]}],"institutions":[],"countries_distinct_count":1,"institutions_distinct_count":1,"corresponding_author_ids":[],"corresponding_institution_ids":[],"apc_list":{"value":3200,"currency":"USD","value_usd":3200},"apc_paid":null,"fwci":null,"has_fulltext":true,"cited_by_count":0,"citation_normalized_percentile":null,"cited_by_percentile_year":null,"biblio":{"volume":"27","issue":"12","first_page":"S549","last_page":"S550"},"is_retracted":false,"is_paratext":false,"is_xpac":false,"primary_topic":{"id":"https://openalex.org/T12400","display_name":"Neurogenetic and Muscular Disorders Research","score":0.5127000212669373,"subfield":{"id":"https://openalex.org/subfields/2716","display_name":"Genetics (clinical)"},"field":{"id":"https://openalex.org/fields/27","display_name":"Medicine"},"domain":{"id":"https://openalex.org/domains/4","display_name":"Health Sciences"}},"topics":[{"id":"https://openalex.org/T12400","display_name":"Neurogenetic and Muscular Disorders Research","score":0.5127000212669373,"subfield":{"id":"https://openalex.org/subfields/2716","display_name":"Genetics (clinical)"},"field":{"id":"https://openalex.org/fields/27","display_name":"Medicine"},"domain":{"id":"https://openalex.org/domains/4","display_name":"Health Sciences"}},{"id":"https://openalex.org/T10441","display_name":"Muscle Physiology and Disorders","score":0.18770000338554382,"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/T10804","display_name":"Health Systems, Economic Evaluations, Quality of Life","score":0.032999999821186066,"subfield":{"id":"https://openalex.org/subfields/2002","display_name":"Economics and Econometrics"},"field":{"id":"https://openalex.org/fields/20","display_name":"Economics, Econometrics and Finance"},"domain":{"id":"https://openalex.org/domains/2","display_name":"Social Sciences"}}],"keywords":[{"id":"https://openalex.org/keywords/patient-reported-outcomes","display_name":"patient-reported outcomes","score":0.8799999952316284},{"id":"https://openalex.org/keywords/promis","display_name":"PROMIS","score":0.9380000233650208},{"id":"https://openalex.org/keywords/natural-history-disease","display_name":"natural history (disease)","score":0.8360000252723694},{"id":"https://openalex.org/keywords/baseline-data","display_name":"baseline data","score":0.4449999928474426},{"id":"https://openalex.org/keywords/myopathy","display_name":"myopathy","score":0.39100000262260437}],"concepts":[{"id":"https://openalex.org/C12725497","wikidata":"https://www.wikidata.org/wiki/Q810247","display_name":"Baseline (sea)","level":2,"score":0.8475425243377686},{"id":"https://openalex.org/C39896193","wikidata":"https://www.wikidata.org/wiki/Q380344","display_name":"Descriptive statistics","level":2,"score":0.5529659390449524},{"id":"https://openalex.org/C163276114","wikidata":"https://www.wikidata.org/wiki/Q484591","display_name":"Natural history","level":2,"score":0.5322437286376953},{"id":"https://openalex.org/C71924100","wikidata":"https://www.wikidata.org/wiki/Q11190","display_name":"Medicine","level":0,"score":0.4410795271396637},{"id":"https://openalex.org/C2776608160","wikidata":"https://www.wikidata.org/wiki/Q4785462","display_name":"Natural (archaeology)","level":2,"score":0.41417649388313293},{"id":"https://openalex.org/C205649164","wikidata":"https://www.wikidata.org/wiki/Q1071","display_name":"Geography","level":0,"score":0.20224741101264954},{"id":"https://openalex.org/C126322002","wikidata":"https://www.wikidata.org/wiki/Q11180","display_name":"Internal medicine","level":1,"score":0.17340150475502014},{"id":"https://openalex.org/C105795698","wikidata":"https://www.wikidata.org/wiki/Q12483","display_name":"Statistics","level":1,"score":0.16119703650474548},{"id":"https://openalex.org/C17744445","wikidata":"https://www.wikidata.org/wiki/Q36442","display_name":"Political science","level":0,"score":0.14541789889335632},{"id":"https://openalex.org/C33923547","wikidata":"https://www.wikidata.org/wiki/Q395","display_name":"Mathematics","level":0,"score":0.06599006056785583},{"id":"https://openalex.org/C166957645","wikidata":"https://www.wikidata.org/wiki/Q23498","display_name":"Archaeology","level":1,"score":0.04413563013076782},{"id":"https://openalex.org/C199539241","wikidata":"https://www.wikidata.org/wiki/Q7748","display_name":"Law","level":1,"score":0.0}],"mesh":[],"locations_count":1,"locations":[{"id":"doi:10.1016/j.jval.2024.10.3462","doi":"https://doi.org/10.1016/j.jval.2024.10.3462","is_oa":true,"landing_page_url":"https://doi.org/10.1016/j.jval.2024.10.3462","pdf_url":"https://www.valueinhealthjournal.com/article/S1098-3015(24)06347-2/pdf","source":{"id":"https://openalex.org/S678965","display_name":"Value in Health","issn_l":"1098-3015","issn":["1098-3015","1524-4733"],"is_oa":false,"is_in_doaj":false,"is_core":true,"listed_in":["abdc-a","anvur-class-a","anvur-scientific","cwts-core","dongbi-a","doyens","jufo-2","ki-jl-2","medline","norway-2","poland-140","russia-white-list-1","vabb-shw"],"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":null,"license_id":null,"version":"publishedVersion","is_accepted":true,"is_published":true,"raw_source_name":"Value in Health","raw_type":"journal-article"}],"best_oa_location":{"id":"doi:10.1016/j.jval.2024.10.3462","doi":"https://doi.org/10.1016/j.jval.2024.10.3462","is_oa":true,"landing_page_url":"https://doi.org/10.1016/j.jval.2024.10.3462","pdf_url":"https://www.valueinhealthjournal.com/article/S1098-3015(24)06347-2/pdf","source":{"id":"https://openalex.org/S678965","display_name":"Value in Health","issn_l":"1098-3015","issn":["1098-3015","1524-4733"],"is_oa":false,"is_in_doaj":false,"is_core":true,"listed_in":["abdc-a","anvur-class-a","anvur-scientific","cwts-core","dongbi-a","doyens","jufo-2","ki-jl-2","medline","norway-2","poland-140","russia-white-list-1","vabb-shw"],"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":null,"license_id":null,"version":"publishedVersion","is_accepted":true,"is_published":true,"raw_source_name":"Value in Health","raw_type":"journal-article"},"sustainable_development_goals":[{"display_name":"Good health and well-being","id":"https://metadata.un.org/sdg/3","score":0.9537000060081482}],"study_designs":[],"awards":[],"funders":[],"has_content":{"grobid_xml":true,"pdf":true},"content_urls":{"pdf":"https://content.openalex.org/works/W4405749685.pdf","grobid_xml":"https://content.openalex.org/works/W4405749685.grobid-xml"},"referenced_works_count":0,"referenced_works":[],"related_works":["https://openalex.org/W4405749696","https://openalex.org/W4224292919","https://openalex.org/W4311341116","https://openalex.org/W2950502319","https://openalex.org/W4307951695","https://openalex.org/W4405749928","https://openalex.org/W4311341342","https://openalex.org/W2916250591","https://openalex.org/W2883038161","https://openalex.org/W2994972606"],"abstract_inverted_index":null,"counts_by_year":[],"updated_date":"2026-11-08T12:12:28.789561","created_date":"2024-12-25T00:00:00"}