{"id":"https://openalex.org/W4413343333","doi":"https://doi.org/10.1002/acn3.70165","title":"Long\u2010Term Evaluation of Givinostat in Duchenne Muscular Dystrophy, and Natural History Comparisons","display_name":"Long\u2010Term Evaluation of Givinostat in Duchenne Muscular Dystrophy, and Natural History Comparisons","publication_year":2025,"publication_date":"2025-08-19","ids":{"openalex":"https://openalex.org/W4413343333","doi":"https://doi.org/10.1002/acn3.70165","pmid":"https://pubmed.ncbi.nlm.nih.gov/40830818","pmcid":"https://www.ncbi.nlm.nih.gov/pmc/articles/12623843"},"language":"en","primary_location":{"id":"doi:10.1002/acn3.70165","doi":"https://doi.org/10.1002/acn3.70165","is_oa":true,"landing_page_url":"https://doi.org/10.1002/acn3.70165","pdf_url":"https://onlinelibrary.wiley.com/doi/pdfdirect/10.1002/acn3.70165","source":{"id":"https://openalex.org/S2764382054","display_name":"Annals of Clinical and Translational Neurology","issn_l":"2328-9503","issn":["2328-9503"],"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-100","russia-white-list-1","vabb-shw"],"host_organization":"https://openalex.org/P4310320595","host_organization_name":"Wiley","host_organization_lineage":["https://openalex.org/P4310320595"],"host_organization_lineage_names":["Wiley"],"type":"journal"},"license":"cc-by","license_id":"https://openalex.org/licenses/cc-by","version":"publishedVersion","is_accepted":true,"is_published":true,"raw_source_name":"Annals of Clinical and Translational Neurology","raw_type":"journal-article"},"volume":null,"type":"article","is_traditional":true,"indexed_in":["crossref","datacite","doaj","pubmed"],"open_access":{"is_oa":true,"oa_status":"gold","oa_url":"https://onlinelibrary.wiley.com/doi/pdfdirect/10.1002/acn3.70165","any_repository_has_fulltext":true},"authorships":[{"author_position":"first","author":{"id":"https://openalex.org/A5071163954","display_name":"Craig M. McDonald","orcid":"https://orcid.org/0000-0002-8779-3220","observed_orcids":["https://orcid.org/0000-0002-8779-3220"]},"institutions":[{"id":"https://openalex.org/I4210160527","display_name":"University of California Davis Medical Center","ror":"https://ror.org/05t6gpm70","country_code":"US","type":"healthcare","lineage":["https://openalex.org/I4210160527","https://openalex.org/I4210160856"]},{"id":"https://openalex.org/I4210160856","display_name":"UC Davis Health","ror":"https://ror.org/05q8kyc69","country_code":"US","type":"healthcare","lineage":["https://openalex.org/I4210160856"]},{"id":"https://openalex.org/I84218800","display_name":"University of California, Davis","ror":"https://ror.org/05rrcem69","country_code":"US","type":"education","lineage":["https://openalex.org/I84218800"]}],"countries":["US"],"is_corresponding":true,"raw_author_name":"Craig M. McDonald","raw_affiliation_strings":["University of California Davis Health, Sacramento, California, USA"],"raw_orcid":null,"affiliations":[{"raw_affiliation_string":"University of California Davis Health, Sacramento, California, USA","institution_ids":["https://openalex.org/I4210160527","https://openalex.org/I4210160856","https://openalex.org/I84218800"]}]},{"author_position":"middle","author":{"id":"https://openalex.org/A5012227405","display_name":"Michela Guglieri","orcid":"https://orcid.org/0000-0002-8455-0637","observed_orcids":["https://orcid.org/0000-0002-8455-0637"]},"institutions":[{"id":"https://openalex.org/I2801663869","display_name":"Newcastle upon Tyne Hospitals NHS Foundation Trust","ror":"https://ror.org/05p40t847","country_code":"GB","type":"healthcare","lineage":["https://openalex.org/I2801663869"]},{"id":"https://openalex.org/I84884186","display_name":"Newcastle University","ror":"https://ror.org/01kj2bm70","country_code":"GB","type":"education","lineage":["https://openalex.org/I84884186"]}],"countries":["GB"],"is_corresponding":false,"raw_author_name":"Michela Guglieri","raw_affiliation_strings":["The John Walton Muscular Dystrophy Research Centre, Translational and Clinical Research Institute, Newcastle University and Newcastle Hospitals NHS Foundation Trust, Newcastle upon Tyne, UK"],"raw_orcid":null,"affiliations":[{"raw_affiliation_string":"The John Walton Muscular Dystrophy Research Centre, Translational and Clinical Research Institute, Newcastle University and Newcastle Hospitals NHS Foundation Trust, Newcastle upon Tyne, UK","institution_ids":["https://openalex.org/I2801663869","https://openalex.org/I84884186"]}]},{"author_position":"middle","author":{"id":"https://openalex.org/A5036524213","display_name":"Dragana Vu\u010dini\u0107","orcid":null,"observed_orcids":[]},"institutions":[],"countries":[],"is_corresponding":false,"raw_author_name":"Dragana Vu\u010dini\u0107","raw_affiliation_strings":["Clinic for Neurology and Psychiatry for Children and Youth, Belgrade, Serbia"],"raw_orcid":null,"affiliations":[{"raw_affiliation_string":"Clinic for Neurology and Psychiatry for Children and Youth, Belgrade, Serbia","institution_ids":[]}]},{"author_position":"middle","author":{"id":"https://openalex.org/A5058740801","display_name":"Gyula Acs\u00e1di","orcid":"https://orcid.org/0000-0001-5408-6362","observed_orcids":["https://orcid.org/0000-0001-5408-6362"]},"institutions":[{"id":"https://openalex.org/I4210101648","display_name":"Connecticut Children's Medical Center","ror":"https://ror.org/01a1jjn24","country_code":"US","type":"healthcare","lineage":["https://openalex.org/I4210101648"]}],"countries":["US"],"is_corresponding":false,"raw_author_name":"Gyula Acsadi","raw_affiliation_strings":["Division of Neurology, Connecticut Children's, Hartford, Connecticut, USA"],"raw_orcid":null,"affiliations":[{"raw_affiliation_string":"Division of Neurology, Connecticut Children's, Hartford, Connecticut, USA","institution_ids":["https://openalex.org/I4210101648"]}]},{"author_position":"middle","author":{"id":"https://openalex.org/A5015956195","display_name":"John Frederick Brandsema","orcid":"https://orcid.org/0000-0001-5945-3280","observed_orcids":["https://orcid.org/0000-0001-5945-3280"]},"institutions":[{"id":"https://openalex.org/I1335321130","display_name":"Children's Hospital of Philadelphia","ror":"https://ror.org/01z7r7q48","country_code":"US","type":"healthcare","lineage":["https://openalex.org/I1335321130"]}],"countries":["US"],"is_corresponding":false,"raw_author_name":"John F. Brandsema","raw_affiliation_strings":["Division of Neurology, The Children's Hospital of Philadelphia, Philadelphia, Pennsylvania, USA"],"raw_orcid":null,"affiliations":[{"raw_affiliation_string":"Division of Neurology, The Children's Hospital of Philadelphia, Philadelphia, Pennsylvania, USA","institution_ids":["https://openalex.org/I1335321130"]}]},{"author_position":"middle","author":{"id":"https://openalex.org/A5103033200","display_name":"Claudio Bruno","orcid":"https://orcid.org/0000-0002-3426-2901","observed_orcids":["https://orcid.org/0000-0002-3426-2901"]},"institutions":[{"id":"https://openalex.org/I2801463291","display_name":"Istituto Giannina Gaslini","ror":"https://ror.org/0424g0k78","country_code":"IT","type":"healthcare","lineage":["https://openalex.org/I2801463291","https://openalex.org/I4210153126"]},{"id":"https://openalex.org/I83816512","display_name":"University of Genoa","ror":"https://ror.org/0107c5v14","country_code":"IT","type":"education","lineage":["https://openalex.org/I83816512"]}],"countries":["IT"],"is_corresponding":false,"raw_author_name":"Claudio Bruno","raw_affiliation_strings":["Center of Translational and Experimental Myology, IRCCS Istituto Giannina Gaslini, Genova, Italy","Department of Neuroscience, Rehabilitation, Ophthalmology, Genetics, Maternal and Child Health \u2013 DINOGMI University of Genova  Genova Italy"],"raw_orcid":null,"affiliations":[{"raw_affiliation_string":"Center of Translational and Experimental Myology, IRCCS Istituto Giannina Gaslini, Genova, Italy","institution_ids":["https://openalex.org/I2801463291"]},{"raw_affiliation_string":"Department of Neuroscience, Rehabilitation, Ophthalmology, Genetics, Maternal and Child Health \u2013 DINOGMI University of Genova  Genova Italy","institution_ids":["https://openalex.org/I83816512"]}]},{"author_position":"middle","author":{"id":"https://openalex.org/A5073529571","display_name":"Erika L. Finanger","orcid":"https://orcid.org/0000-0002-9420-5458","observed_orcids":["https://orcid.org/0000-0002-9420-5458"]},"institutions":[{"id":"https://openalex.org/I2800078164","display_name":"Shriners Hospitals for Children - Portland","ror":"https://ror.org/00snwj435","country_code":"US","type":"healthcare","lineage":["https://openalex.org/I2800078164","https://openalex.org/I4210144267"]}],"countries":["US"],"is_corresponding":false,"raw_author_name":"Erika L. Finanger","raw_affiliation_strings":["Shriners Hospitals for Children \u2013 Portland  Portland Oregon USA"],"raw_orcid":null,"affiliations":[{"raw_affiliation_string":"Shriners Hospitals for Children \u2013 Portland  Portland Oregon USA","institution_ids":["https://openalex.org/I2800078164"]}]},{"author_position":"middle","author":{"id":"https://openalex.org/A5113544995","display_name":"Amy D. Harper","orcid":null,"observed_orcids":[]},"institutions":[{"id":"https://openalex.org/I184840846","display_name":"Virginia Commonwealth University","ror":"https://ror.org/02nkdxk79","country_code":"US","type":"education","lineage":["https://openalex.org/I184840846"]},{"id":"https://openalex.org/I4210166857","display_name":"Children's Hospital of Richmond at VCU","ror":"https://ror.org/05vp5x049","country_code":"US","type":"healthcare","lineage":["https://openalex.org/I4210166857"]},{"id":"https://openalex.org/I72019824","display_name":"Virginia Commonwealth University Medical Center","ror":"https://ror.org/057xmsr27","country_code":"US","type":"healthcare","lineage":["https://openalex.org/I72019824"]}],"countries":["US"],"is_corresponding":false,"raw_author_name":"Amy Harper","raw_affiliation_strings":["Division of Neurology, Virginia Commonwealth University, Children's Hospital of Richmond, Richmond, Virginia, USA"],"raw_orcid":null,"affiliations":[{"raw_affiliation_string":"Division of Neurology, Virginia Commonwealth University, Children's Hospital of Richmond, Richmond, Virginia, USA","institution_ids":["https://openalex.org/I184840846","https://openalex.org/I4210166857","https://openalex.org/I72019824"]}]},{"author_position":"middle","author":{"id":"https://openalex.org/A5015153378","display_name":"Mercedes L\u00f3pez Lobato","orcid":null,"observed_orcids":[]},"institutions":[{"id":"https://openalex.org/I4210153139","display_name":"Hospital Universitario Virgen del Roc\u00edo","ror":"https://ror.org/04vfhnm78","country_code":"ES","type":"healthcare","lineage":["https://openalex.org/I4210153139"]}],"countries":["ES"],"is_corresponding":false,"raw_author_name":"Mercedes Lopez Lobato","raw_affiliation_strings":["Hospital Universitario Virgen del Rocio, Sevilla, Spain"],"raw_orcid":null,"affiliations":[{"raw_affiliation_string":"Hospital Universitario Virgen del Rocio, Sevilla, Spain","institution_ids":["https://openalex.org/I4210153139"]}]},{"author_position":"middle","author":{"id":"https://openalex.org/A5003682249","display_name":"Riccardo Masson","orcid":"https://orcid.org/0000-0002-9311-452X","observed_orcids":["https://orcid.org/0000-0002-9311-452X"]},"institutions":[{"id":"https://openalex.org/I4210164485","display_name":"Fondazione IRCCS Istituto Neurologico Carlo Besta","ror":"https://ror.org/05rbx8m02","country_code":"IT","type":"healthcare","lineage":["https://openalex.org/I4210153126","https://openalex.org/I4210164485"]}],"countries":["IT"],"is_corresponding":false,"raw_author_name":"Riccardo Masson","raw_affiliation_strings":["Developmental Neurology Unit, Fondazione IRCCS Istituto Neurologico Carlo Besta, Milan, Italy"],"raw_orcid":null,"affiliations":[{"raw_affiliation_string":"Developmental Neurology Unit, Fondazione IRCCS Istituto Neurologico Carlo Besta, Milan, Italy","institution_ids":["https://openalex.org/I4210164485"]}]},{"author_position":"middle","author":{"id":"https://openalex.org/A5037815083","display_name":"Nuria Muelas","orcid":"https://orcid.org/0000-0002-2349-7481","observed_orcids":["https://orcid.org/0000-0002-2349-7481"]},"institutions":[{"id":"https://openalex.org/I3019010403","display_name":"Hospital Universitari i Polit\u00e8cnic La Fe","ror":"https://ror.org/01ar2v535","country_code":"ES","type":"healthcare","lineage":["https://openalex.org/I3019010403","https://openalex.org/I4210166036"]},{"id":"https://openalex.org/I4210111366","display_name":"Centre for Biomedical Network Research on Rare Diseases","ror":"https://ror.org/01ygm5w19","country_code":"ES","type":"facility","lineage":["https://openalex.org/I4210111366"]},{"id":"https://openalex.org/I4210166036","display_name":"Instituto de Investigaci\u00f3n Sanitaria La Fe","ror":"https://ror.org/05n7v5997","country_code":"ES","type":"nonprofit","lineage":["https://openalex.org/I4210166036"]}],"countries":["ES"],"is_corresponding":false,"raw_author_name":"Nuria Muelas","raw_affiliation_strings":["Centro de Investigaci\u00f3n Biom\u00e9dica en Red de Enfermedades Raras (CIBERER), U763  Valencia Spain","Neuromuscular Diseases Unit, Neurology Department Hospital Universitari i Polit\u00e8cnic La Fe  Valencia Spain","Neuromuscular Reference Centre, European Reference Network for Neuromuscular Diseases (ERN\u2010EURO\u2010NMD), Neuromuscular and Ataxias Research Group Instituto de Investigaci\u00f3n Sanitaria La Fe  Valencia Spain"],"raw_orcid":null,"affiliations":[{"raw_affiliation_string":"Centro de Investigaci\u00f3n Biom\u00e9dica en Red de Enfermedades Raras (CIBERER), U763  Valencia Spain","institution_ids":["https://openalex.org/I4210111366"]},{"raw_affiliation_string":"Neuromuscular Diseases Unit, Neurology Department Hospital Universitari i Polit\u00e8cnic La Fe  Valencia Spain","institution_ids":["https://openalex.org/I3019010403"]},{"raw_affiliation_string":"Neuromuscular Reference Centre, European Reference Network for Neuromuscular Diseases (ERN\u2010EURO\u2010NMD), Neuromuscular and Ataxias Research Group Instituto de Investigaci\u00f3n Sanitaria La Fe  Valencia Spain","institution_ids":["https://openalex.org/I4210166036"]}]},{"author_position":"middle","author":{"id":"https://openalex.org/A5003721649","display_name":"Francina Munell","orcid":"https://orcid.org/0000-0001-8580-2819","observed_orcids":["https://orcid.org/0000-0001-8580-2819"]},"institutions":[{"id":"https://openalex.org/I4210127641","display_name":"Vall d'Hebron Hospital Universitari","ror":"https://ror.org/03ba28x55","country_code":"ES","type":"healthcare","lineage":["https://openalex.org/I157913058","https://openalex.org/I4210127641","https://openalex.org/I4210153292"]}],"countries":["ES"],"is_corresponding":false,"raw_author_name":"Francina Munell","raw_affiliation_strings":["Pediatric Neuromuscular Diseases Unit, Pediatric Neurology Section, Pediatric Department, Hospital Universitari Vall d'Hebron, European Reference Network for Neuromuscular Diseases (ERN-EURO-NMD), Barcelona, Spain"],"raw_orcid":null,"affiliations":[{"raw_affiliation_string":"Pediatric Neuromuscular Diseases Unit, Pediatric Neurology Section, Pediatric Department, Hospital Universitari Vall d'Hebron, European Reference Network for Neuromuscular Diseases (ERN-EURO-NMD), Barcelona, Spain","institution_ids":["https://openalex.org/I4210127641"]}]},{"author_position":"middle","author":{"id":"https://openalex.org/A5084992423","display_name":"Yoram Nevo","orcid":"https://orcid.org/0000-0002-1434-0976","observed_orcids":["https://orcid.org/0000-0002-1434-0976"]},"institutions":[{"id":"https://openalex.org/I16391192","display_name":"Tel Aviv University","ror":"https://ror.org/04mhzgx49","country_code":"IL","type":"education","lineage":["https://openalex.org/I16391192"]},{"id":"https://openalex.org/I4210109312","display_name":"Schneider Children's Medical Center","ror":"https://ror.org/01z3j3n30","country_code":"IL","type":"healthcare","lineage":["https://openalex.org/I4210109312"]}],"countries":["IL"],"is_corresponding":false,"raw_author_name":"Yoram Nevo","raw_affiliation_strings":["Institute of Neurology \u2013 Schneider Children's Medical Center of Israel Tel\u2010Aviv University  Tel\u2010Aviv Israel"],"raw_orcid":null,"affiliations":[{"raw_affiliation_string":"Institute of Neurology \u2013 Schneider Children's Medical Center of Israel Tel\u2010Aviv University  Tel\u2010Aviv Israel","institution_ids":["https://openalex.org/I16391192","https://openalex.org/I4210109312"]}]},{"author_position":"middle","author":{"id":"https://openalex.org/A5050704861","display_name":"Yann P\u00e9r\u00e9on","orcid":"https://orcid.org/0000-0002-0090-3571","observed_orcids":["https://orcid.org/0000-0002-0090-3571"]},"institutions":[{"id":"https://openalex.org/I3019204325","display_name":"Centre Hospitalier Universitaire de Nantes","ror":"https://ror.org/05c1qsg97","country_code":"FR","type":"healthcare","lineage":["https://openalex.org/I3019204325"]}],"countries":["FR"],"is_corresponding":false,"raw_author_name":"Yann P\u00e9r\u00e9on","raw_affiliation_strings":["CHU Nantes, Reference Centre for Neuromuscular Disorders AOC FILNEMUS, Euro\u2010NMD H\u00f4tel\u2010Dieu  Nantes France"],"raw_orcid":null,"affiliations":[{"raw_affiliation_string":"CHU Nantes, Reference Centre for Neuromuscular Disorders AOC FILNEMUS, Euro\u2010NMD H\u00f4tel\u2010Dieu  Nantes France","institution_ids":["https://openalex.org/I3019204325"]}]},{"author_position":"middle","author":{"id":"https://openalex.org/A5108782387","display_name":"Han C. Phan","orcid":null,"observed_orcids":[]},"institutions":[],"countries":[],"is_corresponding":false,"raw_author_name":"Han Phan","raw_affiliation_strings":["MD Rare Disease Research, LLC, Atlanta, Georgia, USA"],"raw_orcid":null,"affiliations":[{"raw_affiliation_string":"MD Rare Disease Research, LLC, Atlanta, Georgia, USA","institution_ids":[]}]},{"author_position":"middle","author":{"id":"https://openalex.org/A5017152174","display_name":"Valeria Sansone","orcid":"https://orcid.org/0000-0002-1354-3800","observed_orcids":["https://orcid.org/0000-0002-1354-3800"]},"institutions":[{"id":"https://openalex.org/I189158943","display_name":"University of Milan","ror":"https://ror.org/00wjc7c48","country_code":"IT","type":"education","lineage":["https://openalex.org/I189158943"]},{"id":"https://openalex.org/I4210111425","display_name":"Centro Clinico Nemo","ror":"https://ror.org/01xht6a47","country_code":"IT","type":"healthcare","lineage":["https://openalex.org/I4210111425"]}],"countries":["IT"],"is_corresponding":false,"raw_author_name":"Valeria A. Sansone","raw_affiliation_strings":["Neurorehabilitation Unit, The NeMO Clinical Center in Milan, University of Milan, Milan, Italy"],"raw_orcid":null,"affiliations":[{"raw_affiliation_string":"Neurorehabilitation Unit, The NeMO Clinical Center in Milan, University of Milan, Milan, Italy","institution_ids":["https://openalex.org/I189158943","https://openalex.org/I4210111425"]}]},{"author_position":"middle","author":{"id":"https://openalex.org/A5027305120","display_name":"Mariacristina Scoto","orcid":"https://orcid.org/0000-0002-7320-6443","observed_orcids":["https://orcid.org/0000-0002-7320-6443"]},"institutions":[{"id":"https://openalex.org/I2800129641","display_name":"Great Ormond Street Hospital","ror":"https://ror.org/00zn2c847","country_code":"GB","type":"healthcare","lineage":["https://openalex.org/I2800129641","https://openalex.org/I2800349819"]},{"id":"https://openalex.org/I45129253","display_name":"University College London","ror":"https://ror.org/02jx3x895","country_code":"GB","type":"education","lineage":["https://openalex.org/I124357947","https://openalex.org/I45129253"]}],"countries":["GB"],"is_corresponding":false,"raw_author_name":"Mariacristina Scoto","raw_affiliation_strings":["Dubowitz Neuromuscular Centre and MRC Centre for NMD Great Ormond Street Hospital & UCL Great Ormond Street Institute of Child Health London UK"],"raw_orcid":null,"affiliations":[{"raw_affiliation_string":"Dubowitz Neuromuscular Centre and MRC Centre for NMD Great Ormond Street Hospital & UCL Great Ormond Street Institute of Child Health London UK","institution_ids":["https://openalex.org/I2800129641","https://openalex.org/I45129253"]}]},{"author_position":"middle","author":{"id":"https://openalex.org/A5084624493","display_name":"Tracey Anne Willis","orcid":"https://orcid.org/0000-0003-0088-5307","observed_orcids":["https://orcid.org/0000-0003-0088-5307"]},"institutions":[{"id":"https://openalex.org/I2802478169","display_name":"Robert Jones and Agnes Hunt Orthopaedic Hospital","ror":"https://ror.org/030mbcp39","country_code":"GB","type":"healthcare","lineage":["https://openalex.org/I2802478169","https://openalex.org/I4210155739"]},{"id":"https://openalex.org/I4210155739","display_name":"Robert Jones and Agnes Hunt Orthopaedic Hospital NHS Trust","ror":"https://ror.org/049zedh07","country_code":"GB","type":"healthcare","lineage":["https://openalex.org/I4210155739"]}],"countries":["GB"],"is_corresponding":false,"raw_author_name":"Tracey Willis","raw_affiliation_strings":["The Robert Jones and Agnes Hunt Orthopaedic Hospital NHS Foundation Trust, Oswestry, UK"],"raw_orcid":null,"affiliations":[{"raw_affiliation_string":"The Robert Jones and Agnes Hunt Orthopaedic Hospital NHS Foundation Trust, Oswestry, UK","institution_ids":["https://openalex.org/I2802478169","https://openalex.org/I4210155739"]}]},{"author_position":"middle","author":{"id":"https://openalex.org/A5084459237","display_name":"Richard S. Finkel","orcid":"https://orcid.org/0000-0002-9351-7054","observed_orcids":["https://orcid.org/0000-0002-9351-7054"]},"institutions":[{"id":"https://openalex.org/I1313298211","display_name":"St. Jude Children's Research Hospital","ror":"https://ror.org/02r3e0967","country_code":"US","type":"healthcare","lineage":["https://openalex.org/I1313298211","https://openalex.org/I2802152183"]},{"id":"https://openalex.org/I4210157309","display_name":"Nemours Children's Clinic","ror":"https://ror.org/04hma4w69","country_code":"US","type":"healthcare","lineage":["https://openalex.org/I4210108049","https://openalex.org/I4210157309"]}],"countries":["US"],"is_corresponding":false,"raw_author_name":"Richard S. Finkel","raw_affiliation_strings":["Center for Experimental Neurotherapeutics, St. Jude Children's Research Hospital, Memphis, Tennessee, USA","Division of Neurology, Nemours Children's Hospital, Orlando, Florida, USA"],"raw_orcid":null,"affiliations":[{"raw_affiliation_string":"Center for Experimental Neurotherapeutics, St. Jude Children's Research Hospital, Memphis, Tennessee, USA","institution_ids":["https://openalex.org/I1313298211"]},{"raw_affiliation_string":"Division of Neurology, Nemours Children's Hospital, Orlando, Florida, USA","institution_ids":["https://openalex.org/I4210157309"]}]},{"author_position":"middle","author":{"id":"https://openalex.org/A5023983627","display_name":"Krista Vandenborne","orcid":"https://orcid.org/0009-0007-8183-1381","observed_orcids":["https://orcid.org/0009-0007-8183-1381"]},"institutions":[{"id":"https://openalex.org/I2800717037","display_name":"University of Florida Health","ror":"https://ror.org/04tk2gy88","country_code":"US","type":"education","lineage":["https://openalex.org/I2800717037","https://openalex.org/I33213144"]},{"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":"Krista Vandenborne","raw_affiliation_strings":["ImagingDMD and Department of Physical Therapy, University of Florida, Gainesville, Florida, USA"],"raw_orcid":null,"affiliations":[{"raw_affiliation_string":"ImagingDMD and Department of Physical Therapy, University of Florida, Gainesville, Florida, USA","institution_ids":["https://openalex.org/I2800717037","https://openalex.org/I33213144"]}]},{"author_position":"middle","author":{"id":"https://openalex.org/A5017475778","display_name":"Sara Cazzaniga","orcid":"https://orcid.org/0000-0001-6957-7113","observed_orcids":["https://orcid.org/0000-0001-6957-7113"]},"institutions":[{"id":"https://openalex.org/I4210149202","display_name":"Italfarmaco (Italy)","ror":"https://ror.org/05xsppr58","country_code":"IT","type":"company","lineage":["https://openalex.org/I4210149202"]}],"countries":["IT"],"is_corresponding":false,"raw_author_name":"Sara Cazzaniga","raw_affiliation_strings":["Italfarmaco SpA, Milan, Italy"],"raw_orcid":null,"affiliations":[{"raw_affiliation_string":"Italfarmaco SpA, Milan, Italy","institution_ids":["https://openalex.org/I4210149202"]}]},{"author_position":"middle","author":{"id":"https://openalex.org/A5078773291","display_name":"Silvia Montrasio","orcid":null,"observed_orcids":[]},"institutions":[{"id":"https://openalex.org/I4210149202","display_name":"Italfarmaco (Italy)","ror":"https://ror.org/05xsppr58","country_code":"IT","type":"company","lineage":["https://openalex.org/I4210149202"]}],"countries":["IT"],"is_corresponding":false,"raw_author_name":"Silvia Montrasio","raw_affiliation_strings":["Italfarmaco SpA, Milan, Italy"],"raw_orcid":null,"affiliations":[{"raw_affiliation_string":"Italfarmaco SpA, Milan, Italy","institution_ids":["https://openalex.org/I4210149202"]}]},{"author_position":"middle","author":{"id":null,"display_name":"Federica Alessi","orcid":null,"observed_orcids":[]},"institutions":[{"id":"https://openalex.org/I4210149202","display_name":"Italfarmaco (Italy)","ror":"https://ror.org/05xsppr58","country_code":"IT","type":"company","lineage":["https://openalex.org/I4210149202"]}],"countries":["IT"],"is_corresponding":false,"raw_author_name":"Federica Alessi","raw_affiliation_strings":["Italfarmaco SpA, Milan, Italy"],"raw_orcid":null,"affiliations":[{"raw_affiliation_string":"Italfarmaco SpA, Milan, Italy","institution_ids":["https://openalex.org/I4210149202"]}]},{"author_position":"middle","author":{"id":"https://openalex.org/A5086407745","display_name":"Paolo U. Bettica","orcid":"https://orcid.org/0000-0003-2677-3397","observed_orcids":["https://orcid.org/0000-0003-2677-3397"]},"institutions":[{"id":"https://openalex.org/I4210149202","display_name":"Italfarmaco (Italy)","ror":"https://ror.org/05xsppr58","country_code":"IT","type":"company","lineage":["https://openalex.org/I4210149202"]}],"countries":["IT"],"is_corresponding":false,"raw_author_name":"Paolo Bettica","raw_affiliation_strings":["Italfarmaco SpA, Milan, Italy"],"raw_orcid":null,"affiliations":[{"raw_affiliation_string":"Italfarmaco SpA, Milan, Italy","institution_ids":["https://openalex.org/I4210149202"]}]},{"author_position":"middle","author":{"id":"https://openalex.org/A5042269980","display_name":"Eugenio Maria Mercuri","orcid":"https://orcid.org/0000-0002-9851-5365","observed_orcids":["https://orcid.org/0000-0002-9851-5365","https://orcid.org/0000-0002-1005-3443"]},"institutions":[{"id":"https://openalex.org/I103320735","display_name":"Universit\u00e0 Cattolica del Sacro Cuore","ror":"https://ror.org/03h7r5v07","country_code":"IT","type":"education","lineage":["https://openalex.org/I103320735"]},{"id":"https://openalex.org/I2802557943","display_name":"Agostino Gemelli University Polyclinic","ror":"https://ror.org/00rg70c39","country_code":"IT","type":"healthcare","lineage":["https://openalex.org/I2802557943"]},{"id":"https://openalex.org/I4210111425","display_name":"Centro Clinico Nemo","ror":"https://ror.org/01xht6a47","country_code":"IT","type":"healthcare","lineage":["https://openalex.org/I4210111425"]}],"countries":["IT"],"is_corresponding":false,"raw_author_name":"Eugenio Mercuri","raw_affiliation_strings":["Centro Clinico Nemo Fondazione Policlinico Gemelli IRCCS, Rome, Italy","Pediatric Neurology, Universita Cattolica del Sacro Cuore, Rome, Italy"],"raw_orcid":null,"affiliations":[{"raw_affiliation_string":"Centro Clinico Nemo Fondazione Policlinico Gemelli IRCCS, Rome, Italy","institution_ids":["https://openalex.org/I2802557943","https://openalex.org/I4210111425"]},{"raw_affiliation_string":"Pediatric Neurology, Universita Cattolica del Sacro Cuore, Rome, Italy","institution_ids":["https://openalex.org/I103320735"]}]},{"author_position":"middle","author":{"id":"https://openalex.org/A5143235184","display_name":"the Givinostat Study 51 Investigators","orcid":null,"observed_orcids":[]},"institutions":[],"countries":[],"is_corresponding":false,"raw_author_name":"the Givinostat Study 51 Investigators","raw_affiliation_strings":[],"raw_orcid":null,"affiliations":[]},{"author_position":"last","author":{"id":"https://openalex.org/A5143235184","display_name":"the Givinostat Study 51 Investigators","orcid":null,"observed_orcids":[]},"institutions":[],"countries":[],"is_corresponding":false,"raw_author_name":"the ImagingDMD Investigators","raw_affiliation_strings":[],"raw_orcid":null,"affiliations":[]}],"institutions":[],"countries_distinct_count":6,"institutions_distinct_count":35,"corresponding_author_ids":["https://openalex.org/A5071163954"],"corresponding_institution_ids":["https://openalex.org/I4210160527","https://openalex.org/I4210160856","https://openalex.org/I84218800"],"apc_list":{"value":4100,"currency":"USD","value_usd":4100},"apc_paid":{"value":4100,"currency":"USD","value_usd":4100},"fwci":2.87,"has_fulltext":true,"cited_by_count":13,"citation_normalized_percentile":{"value":0.91691732,"is_in_top_1_percent":false,"is_in_top_10_percent":true},"cited_by_percentile_year":{"min":89,"max":99},"biblio":{"volume":"12","issue":"11","first_page":"2335","last_page":"2348"},"is_retracted":false,"is_paratext":false,"is_xpac":false,"primary_topic":{"id":"https://openalex.org/T10441","display_name":"Muscle Physiology and Disorders","score":0.9355000257492065,"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":0.9355000257492065,"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/T12400","display_name":"Neurogenetic and Muscular Disorders Research","score":0.04309999942779541,"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/T10882","display_name":"Cardiomyopathy and Myosin Studies","score":0.0008999999845400453,"subfield":{"id":"https://openalex.org/subfields/2705","display_name":"Cardiology and Cardiovascular Medicine"},"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/givinostat","display_name":"givinostat","score":0.9980000257492065},{"id":"https://openalex.org/keywords/duchenne-muscular-dystrophy","display_name":"Duchenne muscular dystrophy","score":0.9980000257492065},{"id":"https://openalex.org/keywords/histone-deacetylase-inhibitors","display_name":"histone deacetylase inhibitors","score":0.9100000262260437},{"id":"https://openalex.org/keywords/long-term-safety-and-tolerability","display_name":"long-term safety and tolerability","score":0.8980000019073486},{"id":"https://openalex.org/keywords/systemic-corticosteroids","display_name":"systemic corticosteroids","score":0.8669999837875366},{"id":"https://openalex.org/keywords/disease-progression","display_name":"disease progression","score":0.6769999861717224},{"id":"https://openalex.org/keywords/loss-of-ambulation","display_name":"loss of ambulation","score":0.652999997138977},{"id":"https://openalex.org/keywords/stair-climbing-test","display_name":"stair climbing test","score":0.5260000228881836}],"concepts":[{"id":"https://openalex.org/C71924100","wikidata":"https://www.wikidata.org/wiki/Q11190","display_name":"Medicine","level":0,"score":0.8550761938095093},{"id":"https://openalex.org/C2778375690","wikidata":"https://www.wikidata.org/wiki/Q7814214","display_name":"Tolerability","level":3,"score":0.7550292611122131},{"id":"https://openalex.org/C197934379","wikidata":"https://www.wikidata.org/wiki/Q2047938","display_name":"Adverse effect","level":2,"score":0.398100882768631},{"id":"https://openalex.org/C126322002","wikidata":"https://www.wikidata.org/wiki/Q11180","display_name":"Internal medicine","level":1,"score":0.2910487651824951}],"mesh":[{"descriptor_ui":"D000293","descriptor_name":"Adolescent","qualifier_ui":null,"qualifier_name":null,"is_major_topic":false},{"descriptor_ui":"D000328","descriptor_name":"Adult","qualifier_ui":null,"qualifier_name":null,"is_major_topic":false},{"descriptor_ui":"D002219","descriptor_name":"Carbamates","qualifier_ui":"Q000008","qualifier_name":"administration & dosage","is_major_topic":true},{"descriptor_ui":"D002219","descriptor_name":"Carbamates","qualifier_ui":"Q000009","qualifier_name":"adverse effects","is_major_topic":true},{"descriptor_ui":"D002219","descriptor_name":"Carbamates","qualifier_ui":"Q000494","qualifier_name":"pharmacology","is_major_topic":true},{"descriptor_ui":"D002648","descriptor_name":"Child","qualifier_ui":null,"qualifier_name":null,"is_major_topic":false},{"descriptor_ui":"D002675","descriptor_name":"Child, Preschool","qualifier_ui":null,"qualifier_name":null,"is_major_topic":false},{"descriptor_ui":"D005260","descriptor_name":"Female","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":"D008297","descriptor_name":"Male","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},{"descriptor_ui":"D055815","descriptor_name":"Young Adult","qualifier_ui":null,"qualifier_name":null,"is_major_topic":false},{"descriptor_ui":"D056572","descriptor_name":"Histone Deacetylase Inhibitors","qualifier_ui":"Q000008","qualifier_name":"administration & dosage","is_major_topic":true},{"descriptor_ui":"D056572","descriptor_name":"Histone Deacetylase Inhibitors","qualifier_ui":"Q000009","qualifier_name":"adverse effects","is_major_topic":true},{"descriptor_ui":"D056572","descriptor_name":"Histone Deacetylase Inhibitors","qualifier_ui":"Q000494","qualifier_name":"pharmacology","is_major_topic":true}],"locations_count":8,"locations":[{"id":"doi:10.1002/acn3.70165","doi":"https://doi.org/10.1002/acn3.70165","is_oa":true,"landing_page_url":"https://doi.org/10.1002/acn3.70165","pdf_url":"https://onlinelibrary.wiley.com/doi/pdfdirect/10.1002/acn3.70165","source":{"id":"https://openalex.org/S2764382054","display_name":"Annals of Clinical and Translational Neurology","issn_l":"2328-9503","issn":["2328-9503"],"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-100","russia-white-list-1","vabb-shw"],"host_organization":"https://openalex.org/P4310320595","host_organization_name":"Wiley","host_organization_lineage":["https://openalex.org/P4310320595"],"host_organization_lineage_names":["Wiley"],"type":"journal"},"license":"cc-by","license_id":"https://openalex.org/licenses/cc-by","version":"publishedVersion","is_accepted":true,"is_published":true,"raw_source_name":"Annals of Clinical and Translational Neurology","raw_type":"journal-article"},{"id":"pmh:oai:europepmc.org:11432072","doi":null,"is_oa":true,"landing_page_url":"https://www.ncbi.nlm.nih.gov/pmc/articles/12623843","pdf_url":null,"source":{"id":"https://openalex.org/S4306400806","display_name":"Europe PMC (PubMed Central)","issn_l":null,"issn":null,"is_oa":false,"is_in_doaj":false,"is_core":false,"listed_in":[],"host_organization":"https://openalex.org/I1303153112","host_organization_name":"European Bioinformatics Institute","host_organization_lineage":["https://openalex.org/I1303153112"],"host_organization_lineage_names":[],"type":"repository"},"license":"cc-by","license_id":"https://openalex.org/licenses/cc-by","version":"publishedVersion","is_accepted":true,"is_published":true,"raw_source_name":null,"raw_type":"Text"},{"id":"pmid:40830818","doi":null,"is_oa":false,"landing_page_url":"https://pubmed.ncbi.nlm.nih.gov/40830818","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":"Annals of clinical and translational neurology","raw_type":"Clinical Trial, Phase II"},{"id":"pmh:oai:pubmedcentral.nih.gov:12623843","doi":null,"is_oa":true,"landing_page_url":"https://pmc.ncbi.nlm.nih.gov/articles/PMC12623843/","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","license_id":"https://openalex.org/licenses/cc-by","version":"publishedVersion","is_accepted":true,"is_published":true,"raw_source_name":"Ann Clin Transl Neurol","raw_type":"Text"},{"id":"pmh:oai:eprints.ncl.ac.uk:307497","doi":null,"is_oa":true,"landing_page_url":"https://eprints.ncl.ac.uk/fulltext.aspx?url=307497/A017B827-7E8A-4463-8C1F-D462C151BC6D.pdf&pub_id=307497","pdf_url":"https://eprints.ncl.ac.uk/fulltext.aspx?url=307497/A017B827-7E8A-4463-8C1F-D462C151BC6D.pdf&pub_id=307497","source":{"id":"https://openalex.org/S4306402485","display_name":"Newcastle University ePrints (Newcastle Univesity)","issn_l":null,"issn":null,"is_oa":false,"is_in_doaj":false,"is_core":false,"listed_in":[],"host_organization":"https://openalex.org/I84884186","host_organization_name":"Newcastle University","host_organization_lineage":["https://openalex.org/I84884186"],"host_organization_lineage_names":[],"type":"repository"},"license":"cc-by","license_id":"https://openalex.org/licenses/cc-by","version":"submittedVersion","is_accepted":false,"is_published":false,"raw_source_name":"Annals of Clinical and Translational Neurology, 2025","raw_type":"Article"},{"id":"pmh:oai:doaj.org/article:c14ba67597bd4bc9832d7c2acb8866ca","doi":null,"is_oa":true,"landing_page_url":"https://doaj.org/article/c14ba67597bd4bc9832d7c2acb8866ca","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":"Annals of Clinical and Translational Neurology, Vol 12, Iss 11, Pp 2335-2348 (2025)","raw_type":"article"},{"id":"doi:10.25405/ncl.34532748","doi":"https://doi.org/10.25405/ncl.34532748","is_oa":true,"landing_page_url":"https://doi.org/10.25405/ncl.34532748","pdf_url":null,"source":{"id":"https://openalex.org/S4306402485","display_name":"Newcastle University ePrints (Newcastle Univesity)","issn_l":null,"issn":null,"is_oa":false,"is_in_doaj":false,"is_core":false,"listed_in":[],"host_organization":"https://openalex.org/I84884186","host_organization_name":"Newcastle University","host_organization_lineage":["https://openalex.org/I84884186"],"host_organization_lineage_names":[],"type":"repository"},"license":"cc-by","license_id":"https://openalex.org/licenses/cc-by","version":null,"is_accepted":false,"is_published":false,"raw_source_name":null,"raw_type":"JournalArticle"},{"id":"doi:10.25405/ncl.34532748.v1","doi":"https://doi.org/10.25405/ncl.34532748.v1","is_oa":true,"landing_page_url":"https://doi.org/10.25405/ncl.34532748.v1","pdf_url":null,"source":{"id":"https://openalex.org/S4306402485","display_name":"Newcastle University ePrints (Newcastle Univesity)","issn_l":null,"issn":null,"is_oa":false,"is_in_doaj":false,"is_core":false,"listed_in":[],"host_organization":"https://openalex.org/I84884186","host_organization_name":"Newcastle University","host_organization_lineage":["https://openalex.org/I84884186"],"host_organization_lineage_names":[],"type":"repository"},"license":"cc-by","license_id":"https://openalex.org/licenses/cc-by","version":null,"is_accepted":false,"is_published":false,"raw_source_name":null,"raw_type":"JournalArticle"}],"best_oa_location":{"id":"doi:10.1002/acn3.70165","doi":"https://doi.org/10.1002/acn3.70165","is_oa":true,"landing_page_url":"https://doi.org/10.1002/acn3.70165","pdf_url":"https://onlinelibrary.wiley.com/doi/pdfdirect/10.1002/acn3.70165","source":{"id":"https://openalex.org/S2764382054","display_name":"Annals of Clinical and Translational Neurology","issn_l":"2328-9503","issn":["2328-9503"],"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-100","russia-white-list-1","vabb-shw"],"host_organization":"https://openalex.org/P4310320595","host_organization_name":"Wiley","host_organization_lineage":["https://openalex.org/P4310320595"],"host_organization_lineage_names":["Wiley"],"type":"journal"},"license":"cc-by","license_id":"https://openalex.org/licenses/cc-by","version":"publishedVersion","is_accepted":true,"is_published":true,"raw_source_name":"Annals of Clinical and Translational Neurology","raw_type":"journal-article"},"sustainable_development_goals":[{"score":1.0,"display_name":"Good health and well-being","id":"https://metadata.un.org/sdg/3"}],"study_designs":[],"awards":[{"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/F4320328456","display_name":"Italfarmaco","ror":"https://ror.org/05xsppr58"},{"id":"https://openalex.org/F4320332161","display_name":"National Institutes of Health","ror":"https://ror.org/01cwqze88"},{"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":true,"pdf":true},"content_urls":{"pdf":"https://content.openalex.org/works/W4413343333.pdf","grobid_xml":"https://content.openalex.org/works/W4413343333.grobid-xml"},"referenced_works_count":39,"referenced_works":["https://openalex.org/W181967553","https://openalex.org/W1524530894","https://openalex.org/W1592513071","https://openalex.org/W1951817210","https://openalex.org/W1972813632","https://openalex.org/W2041052974","https://openalex.org/W2048470090","https://openalex.org/W2065376245","https://openalex.org/W2088256602","https://openalex.org/W2089484241","https://openalex.org/W2121690051","https://openalex.org/W2126049444","https://openalex.org/W2131850093","https://openalex.org/W2135250056","https://openalex.org/W2169742106","https://openalex.org/W2172191780","https://openalex.org/W2396039069","https://openalex.org/W2402852458","https://openalex.org/W2473278966","https://openalex.org/W2608444169","https://openalex.org/W2735513135","https://openalex.org/W2756371606","https://openalex.org/W2770733887","https://openalex.org/W2784992484","https://openalex.org/W2800992933","https://openalex.org/W2971943538","https://openalex.org/W3036876824","https://openalex.org/W3131782536","https://openalex.org/W3174488138","https://openalex.org/W3176108666","https://openalex.org/W3186033905","https://openalex.org/W3188033198","https://openalex.org/W4281836308","https://openalex.org/W4321496918","https://openalex.org/W4382069925","https://openalex.org/W4392906644","https://openalex.org/W4395033842","https://openalex.org/W4400362765","https://openalex.org/W4408586010"],"related_works":["https://openalex.org/W4414536769","https://openalex.org/W4414823987","https://openalex.org/W4387307351","https://openalex.org/W4408318031","https://openalex.org/W7155979631","https://openalex.org/W4416705596","https://openalex.org/W2884492114","https://openalex.org/W7155037400","https://openalex.org/W4413140929","https://openalex.org/W7156293970"],"abstract_inverted_index":{"OBJECTIVES:":[0],"This":[1],"ongoing,":[2],"open-label":[3,68],"extension":[4],"study":[5],"is":[6],"evaluating":[7],"the":[8,94,118,121,131,148,180,183,188,190,193,197,202,218,245],"long-term":[9,223],"safety,":[10],"tolerability,":[11],"and":[12,19,45,79,147,166,201,220],"efficacy":[13],"of":[14,37,88,101,109,133,151,182,192,204,208,222,225,247],"givinostat,":[15],"a":[16,70,98],"Class":[17],"I":[18],"II":[20],"histone":[21],"deacetylase":[22],"inhibitor,":[23],"in":[24,74,93,106,117,145,169,227],"patients":[25,34,90,134,228],"with":[26,97,155,161,229,233,237],"Duchenne":[27],"muscular":[28],"dystrophy":[29],"(DMD).":[30],"METHODS:":[31],"The":[32],"recruited":[33],"completed":[35],"one":[36,46],"two":[38],"prior":[39,107,119,156],"clinical":[40],"studies":[41],"(one":[42],"Phase":[43,47],"2":[44],"3":[48],"[EPIDYS]),":[49],"receiving":[50],"givinostat":[51,65,102,125,152,173,226,243],"or":[52,54],"placebo,":[53],"were":[55,143],"successfully":[56],"screened":[57],"but":[58],"not":[59],"randomized":[60],"into":[61],"EPIDYS.":[62],"All":[63],"receive":[64],"oral":[66],"suspension":[67],"at":[69],"flexible,":[71],"weight-based":[72],"dose":[73],"addition":[75],"to":[76,124,175,185,195],"systemic":[77,176],"corticosteroids,":[78],"attend":[80],"visits":[81],"every":[82],"4":[83],"months.":[84],"RESULTS:":[85],"A":[86],"total":[87],"194":[89],"are":[91],"included":[92],"current":[95],"analyses,":[96],"mean":[99],"duration":[100],"exposure":[103,123],"(excluding":[104],"use":[105,116],"studies)":[108],"559.6":[110],"days":[111],"(SD":[112],"373.0);":[113],"when":[114],"including":[115],"studies,":[120],"maximum":[122],"was":[126,153,231],">":[127],"8":[128],"years.":[129],"Although":[130],"majority":[132],"reported":[135],"\u2265":[136],"1":[137],"adverse":[138],"event":[139],"(169/194":[140],"[87.1%]),":[141],"most":[142],"mild/moderate":[144],"severity,":[146],"safety":[149,219],"profile":[150],"consistent":[154,232],"studies.":[157,235],"Post":[158],"hoc":[159],"comparisons":[160],"natural":[162,238],"history":[163,239],"datasets":[164],"(ImagingDMD":[165],"CINRG)":[167],"suggest,":[168],"propensity":[170],"matched":[171],"populations,":[172],"added":[174],"corticosteroids":[177],"significantly":[178],"delayed":[179],"loss":[181,191,203],"ability":[184,194],"rise":[186],"from":[187],"floor,":[189],"complete":[196],"4-stair":[198],"climb":[199],"test,":[200],"ambulation":[205],"(by":[206],"medians":[207],"2.0-3.3":[209],"years;":[210],"all":[211],"nominal":[212],"p":[213],"<":[214],"0.05).":[215],"INTERPRETATION:":[216],"Overall,":[217],"tolerability":[221],"administration":[224],"DMD":[230],"previous":[234],"Comparisons":[236],"data":[240],"suggest":[241],"that":[242],"delays":[244],"occurrence":[246],"major":[248],"disease":[249],"progression":[250],"milestones.":[251],"TRIAL":[252],"REGISTRATION:":[253],"EudraCT":[254],"number:":[255],"2017-000397-10;":[256],"ClinicalTrials.gov":[257],"identifier:":[258],"NCT03373968.":[259]},"counts_by_year":[{"year":2026,"cited_by_count":12},{"year":2025,"cited_by_count":1}],"updated_date":"2026-11-10T11:39:43.021295","created_date":"2025-10-10T00:00:00"}