{"id":"https://openalex.org/W4200603057","doi":"https://doi.org/10.1002/psp4.12753","title":"Development of a model\u2010based clinical trial simulation platform to optimize the design of clinical trials for Duchenne muscular dystrophy","display_name":"Development of a model\u2010based clinical trial simulation platform to optimize the design of clinical trials for Duchenne muscular dystrophy","publication_year":2021,"publication_date":"2021-12-08","ids":{"openalex":"https://openalex.org/W4200603057","doi":"https://doi.org/10.1002/psp4.12753","pmid":"https://pubmed.ncbi.nlm.nih.gov/34877803","pmcid":"https://www.ncbi.nlm.nih.gov/pmc/articles/8923721"},"language":"en","primary_location":{"id":"doi:10.1002/psp4.12753","doi":"https://doi.org/10.1002/psp4.12753","is_oa":true,"landing_page_url":"https://doi.org/10.1002/psp4.12753","pdf_url":null,"source":{"id":"https://openalex.org/S4210231731","display_name":"CPT Pharmacometrics & Systems Pharmacology","issn_l":"2163-8306","issn":["2163-8306"],"is_oa":true,"is_in_doaj":true,"is_core":true,"listed_in":["cwts-core","doaj","dongbi-b","doyens","jufo-1","ki-jl-1","medline","norway-1","oarl","poland-70","russia-white-list-1"],"host_organization":"https://openalex.org/P4310319908","host_organization_name":"Nature Portfolio","host_organization_lineage":["https://openalex.org/P4310319908","https://openalex.org/P4310319965"],"host_organization_lineage_names":["Nature Portfolio","Springer Nature"],"type":"journal"},"license":"cc-by-nc","license_id":"https://openalex.org/licenses/cc-by-nc","version":"publishedVersion","is_accepted":true,"is_published":true,"raw_source_name":"CPT: Pharmacometrics & Systems Pharmacology","raw_type":"journal-article"},"volume":null,"type":"article","indexed_in":["crossref","doaj","pubmed"],"open_access":{"is_oa":true,"oa_status":"gold","oa_url":"https://doi.org/10.1002/psp4.12753","any_repository_has_fulltext":true},"authorships":[{"author_position":"first","author":{"id":"https://openalex.org/A5062897128","display_name":"Karthik Lingineni","orcid":"https://orcid.org/0000-0003-1272-8093","observed_orcids":["https://orcid.org/0000-0003-1272-8093"]},"institutions":[{"id":"https://openalex.org/I33213144","display_name":"University of Florida","ror":"https://ror.org/02y3ad647","country_code":"US","type":"education","lineage":["https://openalex.org/I33213144"]}],"countries":["US"],"is_corresponding":false,"raw_author_name":"Karthik Lingineni","raw_affiliation_strings":["Department of Pharmaceutics, Center for Pharmacometrics and Systems Pharmacology, College of Pharmacy, University of Florida, Orlando, Florida, USA"],"raw_orcid":null,"affiliations":[{"raw_affiliation_string":"Department of Pharmaceutics, Center for Pharmacometrics and Systems Pharmacology, College of Pharmacy, University of Florida, Orlando, Florida, USA","institution_ids":["https://openalex.org/I33213144"]}]},{"author_position":"middle","author":{"id":"https://openalex.org/A5020321767","display_name":"Varun Aggarwal","orcid":"https://orcid.org/0000-0002-8406-0961","observed_orcids":["https://orcid.org/0000-0002-8406-0961"]},"institutions":[{"id":"https://openalex.org/I2801686373","display_name":"Critical Path Institute","ror":"https://ror.org/02mgtg880","country_code":"US","type":"nonprofit","lineage":["https://openalex.org/I2801686373"]}],"countries":["US"],"is_corresponding":false,"raw_author_name":"Varun Aggarwal","raw_affiliation_strings":["Critical Path Institute, Tucson, Arizona, USA"],"raw_orcid":"https://orcid.org/0000-0002-8406-0961","affiliations":[{"raw_affiliation_string":"Critical Path Institute, Tucson, Arizona, USA","institution_ids":["https://openalex.org/I2801686373"]}]},{"author_position":"middle","author":{"id":"https://openalex.org/A5074423579","display_name":"Juan Francisco Morales","orcid":"https://orcid.org/0000-0003-1588-9167","observed_orcids":["https://orcid.org/0000-0003-1588-9167"]},"institutions":[{"id":"https://openalex.org/I33213144","display_name":"University of Florida","ror":"https://ror.org/02y3ad647","country_code":"US","type":"education","lineage":["https://openalex.org/I33213144"]}],"countries":["US"],"is_corresponding":false,"raw_author_name":"Juan Francisco Morales","raw_affiliation_strings":["Department of Pharmaceutics, Center for Pharmacometrics and Systems Pharmacology, College of Pharmacy, University of Florida, Orlando, Florida, USA"],"raw_orcid":null,"affiliations":[{"raw_affiliation_string":"Department of Pharmaceutics, Center for Pharmacometrics and Systems Pharmacology, College of Pharmacy, University of Florida, Orlando, Florida, USA","institution_ids":["https://openalex.org/I33213144"]}]},{"author_position":"middle","author":{"id":"https://openalex.org/A5024979557","display_name":"Daniela J. Conrado","orcid":null,"observed_orcids":[]},"institutions":[],"countries":[],"is_corresponding":false,"raw_author_name":"Daniela J. Conrado","raw_affiliation_strings":["e-Quantify LLC, La Jolla, California, USA"],"raw_orcid":null,"affiliations":[{"raw_affiliation_string":"e-Quantify LLC, La Jolla, California, USA","institution_ids":[]}]},{"author_position":"middle","author":{"id":"https://openalex.org/A5034321595","display_name":"Diane Corey","orcid":null,"observed_orcids":[]},"institutions":[{"id":"https://openalex.org/I2801686373","display_name":"Critical Path Institute","ror":"https://ror.org/02mgtg880","country_code":"US","type":"nonprofit","lineage":["https://openalex.org/I2801686373"]}],"countries":["US"],"is_corresponding":false,"raw_author_name":"Diane Corey","raw_affiliation_strings":["Critical Path Institute, Tucson, Arizona, USA"],"raw_orcid":null,"affiliations":[{"raw_affiliation_string":"Critical Path Institute, Tucson, Arizona, USA","institution_ids":["https://openalex.org/I2801686373"]}]},{"author_position":"middle","author":{"id":"https://openalex.org/A5010799250","display_name":"Camille Vong","orcid":"https://orcid.org/0000-0002-9390-2710","observed_orcids":["https://orcid.org/0000-0002-9390-2710"]},"institutions":[{"id":"https://openalex.org/I180857899","display_name":"Pfizer (United States)","ror":"https://ror.org/01xdqrp08","country_code":"US","type":"company","lineage":["https://openalex.org/I180857899"]}],"countries":["US"],"is_corresponding":false,"raw_author_name":"Camille Vong","raw_affiliation_strings":["Global Product Development, Pfizer Inc, Cambridge, Massachusetts, USA"],"raw_orcid":null,"affiliations":[{"raw_affiliation_string":"Global Product Development, Pfizer Inc, Cambridge, Massachusetts, USA","institution_ids":["https://openalex.org/I180857899"]}]},{"author_position":"middle","author":{"id":"https://openalex.org/A5050176243","display_name":"Jackson K. Burton","orcid":"https://orcid.org/0000-0002-0910-2041","observed_orcids":["https://orcid.org/0000-0002-0910-2041"]},"institutions":[{"id":"https://openalex.org/I2801686373","display_name":"Critical Path Institute","ror":"https://ror.org/02mgtg880","country_code":"US","type":"nonprofit","lineage":["https://openalex.org/I2801686373"]}],"countries":["US"],"is_corresponding":false,"raw_author_name":"Jackson Burton","raw_affiliation_strings":["Critical Path Institute, Tucson, Arizona, USA"],"raw_orcid":null,"affiliations":[{"raw_affiliation_string":"Critical Path Institute, Tucson, Arizona, USA","institution_ids":["https://openalex.org/I2801686373"]}]},{"author_position":"middle","author":{"id":"https://openalex.org/A5084106931","display_name":"Jane Larkindale","orcid":"https://orcid.org/0000-0003-1055-9679","observed_orcids":["https://orcid.org/0000-0003-1055-9679"]},"institutions":[{"id":"https://openalex.org/I2801686373","display_name":"Critical Path Institute","ror":"https://ror.org/02mgtg880","country_code":"US","type":"nonprofit","lineage":["https://openalex.org/I2801686373"]}],"countries":["US"],"is_corresponding":false,"raw_author_name":"Jane Larkindale","raw_affiliation_strings":["Critical Path Institute, Tucson, Arizona, USA"],"raw_orcid":"https://orcid.org/0000-0003-1055-9679","affiliations":[{"raw_affiliation_string":"Critical Path Institute, Tucson, Arizona, USA","institution_ids":["https://openalex.org/I2801686373"]}]},{"author_position":"middle","author":{"id":"https://openalex.org/A5058044307","display_name":"Klaus Romero","orcid":"https://orcid.org/0000-0002-8576-8638","observed_orcids":["https://orcid.org/0000-0002-8576-8638"]},"institutions":[{"id":"https://openalex.org/I2801686373","display_name":"Critical Path Institute","ror":"https://ror.org/02mgtg880","country_code":"US","type":"nonprofit","lineage":["https://openalex.org/I2801686373"]}],"countries":["US"],"is_corresponding":false,"raw_author_name":"Klaus Romero","raw_affiliation_strings":["Critical Path Institute, Tucson, Arizona, USA"],"raw_orcid":null,"affiliations":[{"raw_affiliation_string":"Critical Path Institute, Tucson, Arizona, USA","institution_ids":["https://openalex.org/I2801686373"]}]},{"author_position":"middle","author":{"id":"https://openalex.org/A5062673118","display_name":"Stephan Schmidt","orcid":"https://orcid.org/0000-0002-4998-1167","observed_orcids":["https://orcid.org/0000-0002-4998-1167"]},"institutions":[{"id":"https://openalex.org/I33213144","display_name":"University of Florida","ror":"https://ror.org/02y3ad647","country_code":"US","type":"education","lineage":["https://openalex.org/I33213144"]}],"countries":["US"],"is_corresponding":false,"raw_author_name":"Stephan Schmidt","raw_affiliation_strings":["Department of Pharmaceutics, Center for Pharmacometrics and Systems Pharmacology, College of Pharmacy, University of Florida, Orlando, Florida, USA"],"raw_orcid":"https://orcid.org/0000-0002-4998-1167","affiliations":[{"raw_affiliation_string":"Department of Pharmaceutics, Center for Pharmacometrics and Systems Pharmacology, College of Pharmacy, University of Florida, Orlando, Florida, USA","institution_ids":["https://openalex.org/I33213144"]}]},{"author_position":"last","author":{"id":"https://openalex.org/A5100654651","display_name":"Sarah Kim","orcid":"https://orcid.org/0000-0002-9179-0735","observed_orcids":["https://orcid.org/0000-0002-9179-0735","https://orcid.org/0009-0000-4001-2899"]},"institutions":[{"id":"https://openalex.org/I33213144","display_name":"University of Florida","ror":"https://ror.org/02y3ad647","country_code":"US","type":"education","lineage":["https://openalex.org/I33213144"]}],"countries":["US"],"is_corresponding":true,"raw_author_name":"Sarah Kim","raw_affiliation_strings":["Department of Pharmaceutics, Center for Pharmacometrics and Systems Pharmacology, College of Pharmacy, University of Florida, Orlando, Florida, USA"],"raw_orcid":"https://orcid.org/0000-0002-9179-0735","affiliations":[{"raw_affiliation_string":"Department of Pharmaceutics, Center for Pharmacometrics and Systems Pharmacology, College of Pharmacy, University of Florida, Orlando, Florida, USA","institution_ids":["https://openalex.org/I33213144"]}]}],"institutions":[],"countries_distinct_count":1,"institutions_distinct_count":3,"corresponding_author_ids":["https://openalex.org/A5100654651"],"corresponding_institution_ids":["https://openalex.org/I33213144"],"apc_list":{"value":3000,"currency":"USD","value_usd":3000},"apc_paid":{"value":3000,"currency":"USD","value_usd":3000},"fwci":4.0892,"has_fulltext":false,"cited_by_count":28,"citation_normalized_percentile":{"value":0.95859582,"is_in_top_1_percent":false,"is_in_top_10_percent":true},"cited_by_percentile_year":{"min":88,"max":99},"biblio":{"volume":"11","issue":"3","first_page":"318","last_page":"332"},"is_retracted":false,"is_paratext":false,"is_xpac":false,"primary_topic":{"id":"https://openalex.org/T11235","display_name":"Statistical Methods in Clinical Trials","score":0.9721999764442444,"subfield":{"id":"https://openalex.org/subfields/2613","display_name":"Statistics and Probability"},"field":{"id":"https://openalex.org/fields/26","display_name":"Mathematics"},"domain":{"id":"https://openalex.org/domains/3","display_name":"Physical Sciences"}},"topics":[{"id":"https://openalex.org/T11235","display_name":"Statistical Methods in Clinical Trials","score":0.9721999764442444,"subfield":{"id":"https://openalex.org/subfields/2613","display_name":"Statistics and Probability"},"field":{"id":"https://openalex.org/fields/26","display_name":"Mathematics"},"domain":{"id":"https://openalex.org/domains/3","display_name":"Physical Sciences"}},{"id":"https://openalex.org/T12547","display_name":"Pharmaceutical studies and practices","score":0.01899999938905239,"subfield":{"id":"https://openalex.org/subfields/2735","display_name":"Pediatrics, Perinatology and Child Health"},"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/T12400","display_name":"Neurogenetic and Muscular Disorders Research","score":0.002099999925121665,"subfield":{"id":"https://openalex.org/subfields/2716","display_name":"Genetics"},"field":{"id":"https://openalex.org/fields/27","display_name":"Medicine"},"domain":{"id":"https://openalex.org/domains/4","display_name":"Health Sciences"}}],"keywords":[{"id":"https://openalex.org/keywords/duchenne-muscular-dystrophy","display_name":"Duchenne muscular dystrophy","score":1.0},{"id":"https://openalex.org/keywords/clinical-trial-simulation","display_name":"clinical trial simulation","score":0.9300000071525574},{"id":"https://openalex.org/keywords/disease-progression-modeling","display_name":"disease progression modeling","score":0.8809999823570251},{"id":"https://openalex.org/keywords/nonlinear-mixed-effects-models","display_name":"nonlinear mixed-effects models","score":0.8379999995231628},{"id":"https://openalex.org/keywords/forced-vital-capacity","display_name":"forced vital capacity","score":0.9850000143051147},{"id":"https://openalex.org/keywords/clinical-trial-eligibility-criteria","display_name":"inclusion and exclusion criteria","score":0.6489999890327454},{"id":"https://openalex.org/keywords/trial-design-optimization","display_name":"trial design optimization","score":0.6919999718666077},{"id":"https://openalex.org/keywords/regulatory-science","display_name":"regulatory science","score":0.4189999997615814}],"concepts":[{"id":"https://openalex.org/C535046627","wikidata":"https://www.wikidata.org/wiki/Q30612","display_name":"Clinical trial","level":2,"score":0.7902038097381592},{"id":"https://openalex.org/C2778943923","wikidata":"https://www.wikidata.org/wiki/Q1648484","display_name":"Duchenne muscular dystrophy","level":2,"score":0.6281077861785889},{"id":"https://openalex.org/C71924100","wikidata":"https://www.wikidata.org/wiki/Q11190","display_name":"Medicine","level":0,"score":0.528956413269043},{"id":"https://openalex.org/C2779134260","wikidata":"https://www.wikidata.org/wiki/Q12136","display_name":"Disease","level":2,"score":0.47860807180404663},{"id":"https://openalex.org/C148482608","wikidata":"https://www.wikidata.org/wiki/Q17007018","display_name":"Clinical study design","level":3,"score":0.474020779132843},{"id":"https://openalex.org/C99508421","wikidata":"https://www.wikidata.org/wiki/Q2678675","display_name":"Physical medicine and rehabilitation","level":1,"score":0.4648524522781372},{"id":"https://openalex.org/C1862650","wikidata":"https://www.wikidata.org/wiki/Q186005","display_name":"Physical therapy","level":1,"score":0.37278279662132263},{"id":"https://openalex.org/C126322002","wikidata":"https://www.wikidata.org/wiki/Q11180","display_name":"Internal medicine","level":1,"score":0.12491798400878906}],"mesh":[{"descriptor_ui":"D002648","descriptor_name":"Child","qualifier_ui":null,"qualifier_name":null,"is_major_topic":false},{"descriptor_ui":"D003198","descriptor_name":"Computer Simulation","qualifier_ui":null,"qualifier_name":null,"is_major_topic":false},{"descriptor_ui":"D006801","descriptor_name":"Humans","qualifier_ui":null,"qualifier_name":null,"is_major_topic":false},{"descriptor_ui":"D014797","descriptor_name":"Vital Capacity","qualifier_ui":null,"qualifier_name":null,"is_major_topic":false},{"descriptor_ui":"D018450","descriptor_name":"Disease Progression","qualifier_ui":null,"qualifier_name":null,"is_major_topic":false},{"descriptor_ui":"D020388","descriptor_name":"Muscular Dystrophy, Duchenne","qualifier_ui":"Q000188","qualifier_name":"drug therapy","is_major_topic":true}],"locations_count":6,"locations":[{"id":"doi:10.1002/psp4.12753","doi":"https://doi.org/10.1002/psp4.12753","is_oa":true,"landing_page_url":"https://doi.org/10.1002/psp4.12753","pdf_url":null,"source":{"id":"https://openalex.org/S4210231731","display_name":"CPT Pharmacometrics & Systems Pharmacology","issn_l":"2163-8306","issn":["2163-8306"],"is_oa":true,"is_in_doaj":true,"is_core":true,"listed_in":["cwts-core","doaj","dongbi-b","doyens","jufo-1","ki-jl-1","medline","norway-1","oarl","poland-70","russia-white-list-1"],"host_organization":"https://openalex.org/P4310319908","host_organization_name":"Nature Portfolio","host_organization_lineage":["https://openalex.org/P4310319908","https://openalex.org/P4310319965"],"host_organization_lineage_names":["Nature Portfolio","Springer Nature"],"type":"journal"},"license":"cc-by-nc","license_id":"https://openalex.org/licenses/cc-by-nc","version":"publishedVersion","is_accepted":true,"is_published":true,"raw_source_name":"CPT: Pharmacometrics & Systems Pharmacology","raw_type":"journal-article"},{"id":"pmh:oai:pubmedcentral.nih.gov:8923721","doi":null,"is_oa":true,"landing_page_url":"https://www.ncbi.nlm.nih.gov/pmc/articles/8923721","pdf_url":null,"source":{"id":"https://openalex.org/S2764455111","display_name":"PubMed Central","issn_l":null,"issn":null,"is_oa":true,"is_in_doaj":false,"is_core":false,"listed_in":[],"host_organization":"https://openalex.org/I1299303238","host_organization_name":"National Institutes of Health","host_organization_lineage":["https://openalex.org/I1299303238"],"host_organization_lineage_names":[],"type":"repository"},"license":"cc-by-nc","license_id":"https://openalex.org/licenses/cc-by-nc","version":"publishedVersion","is_accepted":true,"is_published":true,"raw_source_name":"CPT Pharmacometrics Syst Pharmacol","raw_type":"Text"},{"id":"pmid:34877803","doi":null,"is_oa":false,"landing_page_url":"https://pubmed.ncbi.nlm.nih.gov/34877803","pdf_url":null,"source":{"id":"https://openalex.org/S4306525036","display_name":"PubMed","issn_l":null,"issn":null,"is_oa":false,"is_in_doaj":false,"is_core":false,"listed_in":[],"host_organization":"https://openalex.org/I1299303238","host_organization_name":"National Institutes of Health","host_organization_lineage":["https://openalex.org/I1299303238"],"host_organization_lineage_names":[],"type":"repository"},"license":null,"license_id":null,"version":"publishedVersion","is_accepted":true,"is_published":true,"raw_source_name":"CPT: pharmacometrics & systems pharmacology","raw_type":"Journal Article"},{"id":"pmh:oai:doaj.org/article:9a5c7e29fe9d4a999c3b88d80d68d01e","doi":null,"is_oa":true,"landing_page_url":"https://doaj.org/article/9a5c7e29fe9d4a999c3b88d80d68d01e","pdf_url":null,"source":{"id":"https://openalex.org/S4306401280","display_name":"DOAJ (DOAJ: Directory of Open Access Journals)","issn_l":null,"issn":null,"is_oa":false,"is_in_doaj":false,"is_core":false,"listed_in":[],"host_organization":null,"host_organization_name":null,"host_organization_lineage":[],"host_organization_lineage_names":[],"type":"repository"},"license":"cc-by-sa","license_id":"https://openalex.org/licenses/cc-by-sa","version":"submittedVersion","is_accepted":false,"is_published":false,"raw_source_name":"CPT: Pharmacometrics & Systems Pharmacology, Vol 11, Iss 3, Pp 318-332 (2022)","raw_type":"article"},{"id":"pmh:oai:escholarship.org:ark:/13030/qt3bk5j4rs","doi":null,"is_oa":true,"landing_page_url":"https://escholarship.org/uc/item/3bk5j4rs","pdf_url":null,"source":{"id":"https://openalex.org/S4306400115","display_name":"eScholarship (California Digital Library)","issn_l":null,"issn":null,"is_oa":false,"is_in_doaj":false,"is_core":false,"listed_in":[],"host_organization":"https://openalex.org/I2801248553","host_organization_name":"California Digital Library","host_organization_lineage":["https://openalex.org/I2801248553"],"host_organization_lineage_names":[],"type":"repository"},"license":"cc-by-nc","license_id":"https://openalex.org/licenses/cc-by-nc","version":"submittedVersion","is_accepted":false,"is_published":false,"raw_source_name":"CPT Pharmacometrics & Systems Pharmacology, vol 11, iss 3","raw_type":"article"},{"id":"pmh:oai:jupiter.its.unimelb.edu.au:11343/301463","doi":null,"is_oa":true,"landing_page_url":"https://hdl.handle.net/11343/301463","pdf_url":null,"source":{"id":"https://openalex.org/S4377196259","display_name":"Minerva Access (University of Melbourne)","issn_l":null,"issn":null,"is_oa":false,"is_in_doaj":false,"is_core":false,"listed_in":[],"host_organization":"https://openalex.org/I165779595","host_organization_name":"The University of Melbourne","host_organization_lineage":["https://openalex.org/I165779595"],"host_organization_lineage_names":[],"type":"repository"},"license":"cc-by-nc","license_id":"https://openalex.org/licenses/cc-by-nc","version":"submittedVersion","is_accepted":false,"is_published":false,"raw_source_name":null,"raw_type":"Journal Article"}],"best_oa_location":{"id":"doi:10.1002/psp4.12753","doi":"https://doi.org/10.1002/psp4.12753","is_oa":true,"landing_page_url":"https://doi.org/10.1002/psp4.12753","pdf_url":null,"source":{"id":"https://openalex.org/S4210231731","display_name":"CPT Pharmacometrics & Systems Pharmacology","issn_l":"2163-8306","issn":["2163-8306"],"is_oa":true,"is_in_doaj":true,"is_core":true,"listed_in":["cwts-core","doaj","dongbi-b","doyens","jufo-1","ki-jl-1","medline","norway-1","oarl","poland-70","russia-white-list-1"],"host_organization":"https://openalex.org/P4310319908","host_organization_name":"Nature Portfolio","host_organization_lineage":["https://openalex.org/P4310319908","https://openalex.org/P4310319965"],"host_organization_lineage_names":["Nature Portfolio","Springer Nature"],"type":"journal"},"license":"cc-by-nc","license_id":"https://openalex.org/licenses/cc-by-nc","version":"publishedVersion","is_accepted":true,"is_published":true,"raw_source_name":"CPT: Pharmacometrics & Systems Pharmacology","raw_type":"journal-article"},"sustainable_development_goals":[{"id":"https://metadata.un.org/sdg/3","score":1.0,"display_name":"Good health and well-being"}],"sustainable_development_goals_aurora":[{"id":"https://metadata.un.org/sdg/10","score":0.5799999833106995,"display_name":"Reduced inequalities"}],"study_designs":[],"awards":[{"id":"https://openalex.org/G1747133292","display_name":null,"funder_award_id":"U18FD005320","funder_id":"https://openalex.org/F4320332163","funder_display_name":"U.S. Food and Drug Administration"},{"id":"https://openalex.org/G2370984598","display_name":"Clinically Meaningful Outcomes for Duchenne Muscular Dystrophy Therapeutic Trials","funder_award_id":"R01AR061875","funder_id":"https://openalex.org/F4320337362","funder_display_name":"National Institute of Arthritis and Musculoskeletal and Skin Diseases"},{"id":"https://openalex.org/G8877263756","display_name":"Magnetic Resonance Imaging and Biomarkers for Muscular Dystrophy","funder_award_id":"R01AR056973","funder_id":"https://openalex.org/F4320337362","funder_display_name":"National Institute of Arthritis and Musculoskeletal and Skin Diseases"}],"funders":[{"id":"https://openalex.org/F4320332163","display_name":"U.S. Food and Drug Administration","ror":"https://ror.org/034xvzb47"},{"id":"https://openalex.org/F4320337362","display_name":"National Institute of Arthritis and Musculoskeletal and Skin Diseases","ror":"https://ror.org/006zn3t30"}],"has_content":{"grobid_xml":false,"pdf":false},"content_urls":null,"referenced_works_count":27,"referenced_works":["https://openalex.org/W89647785","https://openalex.org/W1814931903","https://openalex.org/W1855988937","https://openalex.org/W2039192863","https://openalex.org/W2045144063","https://openalex.org/W2105034643","https://openalex.org/W2114123573","https://openalex.org/W2134560161","https://openalex.org/W2137703332","https://openalex.org/W2154041187","https://openalex.org/W2296709708","https://openalex.org/W2337480016","https://openalex.org/W2608444169","https://openalex.org/W2644551405","https://openalex.org/W2756371606","https://openalex.org/W2765458529","https://openalex.org/W2784397775","https://openalex.org/W2784812244","https://openalex.org/W2784992484","https://openalex.org/W2791293365","https://openalex.org/W2800992933","https://openalex.org/W2808493667","https://openalex.org/W2945724922","https://openalex.org/W2948019101","https://openalex.org/W2954356167","https://openalex.org/W3044932657","https://openalex.org/W4200603057"],"related_works":["https://openalex.org/W4387310712","https://openalex.org/W4404768716","https://openalex.org/W2977177960","https://openalex.org/W3090659200","https://openalex.org/W4318671203","https://openalex.org/W4403113261","https://openalex.org/W4385514244","https://openalex.org/W2587562286","https://openalex.org/W4407699873","https://openalex.org/W7131280460"],"abstract_inverted_index":{"Early":[0],"clinical":[1,32,52,60,92],"trials":[2,53,93],"of":[3,25,37,40,50,108,122,171,200,204],"therapies":[4],"to":[5,46,130,135,192,196],"treat":[6],"Duchenne":[7,81],"muscular":[8],"dystrophy":[9],"(DMD),":[10],"a":[11,56,101,169],"fatal":[12],"genetic":[13],"X-linked":[14],"pediatric":[15],"disease,":[16],"have":[17,215,230],"been":[18,216,231],"designed":[19],"based":[20,65],"on":[21,66,148],"the":[22,38,41,48,80,87,120,123,157,176,189,198,219,226,234],"limited":[23],"understanding":[24],"natural":[26],"disease":[27,57,164],"progression":[28,58],"and":[29,94,119,139,152,173,175,206,213,222,225,236,243],"variability":[30],"in":[31,70,156,178,180,188],"measures":[33,67,110,159],"over":[34],"different":[35],"stages":[36],"continuum":[39],"disease.":[42],"The":[43,144,183,210],"objective":[44],"was":[45],"inform":[47],"design":[49],"DMD":[51,71],"by":[54,86,218,250],"developing":[55],"model-based":[59],"trial":[61,194,208],"simulation":[62],"(CTS)":[63],"platform":[64,191],"commonly":[68],"used":[69,187],"trials.":[72],"Data":[73],"were":[74,111,146],"integrated":[75],"from":[76,132],"past":[77],"studies":[78],"through":[79],"Regulatory":[82],"Science":[83],"Consortium":[84],"founded":[85],"Critical":[88],"Path":[89],"Institute":[90],"(15":[91],"studies,":[95],"1505":[96],"subjects,":[97],"27,252":[98],"observations).":[99],"Using":[100],"nonlinear":[102],"mixed-effects":[103],"modeling":[104],"approach,":[105],"longitudinal":[106,154],"dynamics":[107],"five":[109,158],"modeled":[112],"(NorthStar":[113],"Ambulatory":[114],"Assessment,":[115],"forced":[116],"vital":[117],"capacity,":[118],"velocities":[121],"following":[124],"three":[125],"timed":[126],"functional":[127],"tests:":[128],"time":[129,134],"stand":[131],"supine,":[133],"climb":[136],"4":[137],"stairs,":[138],"10":[140],"meter":[141],"walk-run":[142],"time).":[143],"models":[145,184,214],"validated":[147],"external":[149],"data":[150,211],"sets":[151,212],"captured":[153],"changes":[155],"well,":[160],"including":[161],"both":[162,251],"early":[163],"when":[165],"function":[166,179],"improves":[167],"as":[168],"result":[170],"growth":[172],"development":[174],"decline":[177],"later":[181],"stages.":[182],"can":[185],"be":[186,245],"CTS":[190],"perform":[193],"simulations":[195],"optimize":[197],"selection":[199,203],"inclusion/exclusion":[201],"criteria,":[202],"measures,":[205],"other":[207],"parameters.":[209],"reviewed":[217],"US":[220],"Food":[221],"Drug":[223],"Administration":[224],"European":[227],"Medicines":[228],"Agency;":[229],"accepted":[232],"into":[233],"Fit-for-Purpose":[235],"Qualification":[237],"for":[238,247],"Novel":[239],"Methodologies":[240],"pathways,":[241],"respectively;":[242],"will":[244],"submitted":[246],"potential":[248],"endorsement":[249],"agencies.":[252]},"counts_by_year":[{"year":2026,"cited_by_count":1},{"year":2025,"cited_by_count":5},{"year":2024,"cited_by_count":11},{"year":2023,"cited_by_count":7},{"year":2022,"cited_by_count":3},{"year":2021,"cited_by_count":1}],"updated_date":"2026-10-31T12:09:11.096373","created_date":"2021-12-31T00:00:00"}