{"status":"ok","message-type":"work","message-version":"1.0.0","message":{"indexed":{"date-parts":[[2026,9,1]],"date-time":"2026-09-01T07:15:10Z","timestamp":1788246910746,"version":"build-2803163510"},"reference-count":78,"publisher":"Wiley","license":[{"start":{"date-parts":[[2026,8,31]],"date-time":"2026-08-31T00:00:00Z","timestamp":1788134400000},"content-version":"vor","delay-in-days":0,"URL":"http:\/\/onlinelibrary.wiley.com\/termsAndConditions#vor"},{"start":{"date-parts":[[2026,8,31]],"date-time":"2026-08-31T00:00:00Z","timestamp":1788134400000},"content-version":"tdm","delay-in-days":0,"URL":"http:\/\/doi.wiley.com\/10.1002\/tdm_license_1.1"}],"content-domain":{"domain":["onlinelibrary.wiley.com"],"crossmark-restriction":true},"short-container-title":["Haemophilia"],"abstract":"<jats:title>ABSTRACT<\/jats:title>\n                  <jats:sec>\n                    <jats:title>Introduction and Objectives<\/jats:title>\n                    <jats:p>Hemophilia management has evolved from conventional factor replacement to non\u2010factor therapies and gene therapy. However, most clinical trials of modern therapies are conducted in high\u2010 and upper\u2010middle\u2010income countries, with limited participation from low\u2010 and middle\u2010income regions. This disparity reflects broader inequities in access to advanced treatments. This study summarizes the clinical development timeline, regulatory status, methodological approaches, and global distribution of clinical trials on modern hemophilia therapies.<\/jats:p>\n                  <\/jats:sec>\n                  <jats:sec>\n                    <jats:title>Methods<\/jats:title>\n                    <jats:p>A PRISMA\u2010guided systematic review was conducted using PubMed and EBSCO databases. Clinical trials evaluating gene and non\u2010factor therapies were identified through structured keyword searches. Titles, abstracts, and full texts were screened using predefined criteria. Data were synthesized to describe clinical progress, approval status, and geographic participation. Manual reference screening and citation tracking were also performed.<\/jats:p>\n                  <\/jats:sec>\n                  <jats:sec>\n                    <jats:title>Results<\/jats:title>\n                    <jats:p>Identified agents included gene therapies (valoctocogene roxaparvovec, etranacogene dezaparvovec; late\u2010stage fidanacogene elaparvovec), siRNA therapy (fitusiran), anti\u2010TFPI antibodies (concizumab, marstacimab), and the FVIII\u2010mimetic antibody emicizumab. Gene therapy is dominated by AAV platforms, offering durable expression but limited by immunogenicity, re\u2010dosing constraints, and high costs. SiRNA and anti\u2010TFPI therapies enhance thrombin generation but require careful dosing to mitigate thrombotic risk. Emicizumab provides convenient subcutaneous prophylaxis but remains costly with limited data in pediatric and resource\u2010limited settings. Most trials were conducted in high\u2010income countries, with minimal LMIC representation.<\/jats:p>\n                  <\/jats:sec>\n                  <jats:sec>\n                    <jats:title>Conclusion<\/jats:title>\n                    <jats:p>Modern therapies demonstrate strong efficacy and acceptable safety, with several approved agents. However, persistent global inequities in trial participation and access highlight the need for more inclusive research and equitable implementation strategies.<\/jats:p>\n                  <\/jats:sec>","DOI":"10.1111\/hae.70397","type":"journal-article","created":{"date-parts":[[2026,9,1]],"date-time":"2026-09-01T06:39:51Z","timestamp":1788244791000},"update-policy":"https:\/\/doi.org\/10.1002\/crossmark_policy","source":"Crossref","is-referenced-by-count":0,"title":["Bridging the Gap: A Systematic Review of Modern Hemophilia Therapies and Global Inequities in Clinical Trial Participation"],"prefix":"10.1111","author":[{"given":"Nadiya Nurul","family":"Afifah","sequence":"first","affiliation":[{"name":"Universitas Esa Unggul  Jakarta Indonesia"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Lanny Indah","family":"Permatasari","sequence":"additional","affiliation":[{"name":"Universitas An Nasher  Cirebon Indonesia"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Irma Rahayu","family":"Latarissa","sequence":"additional","affiliation":[{"name":"Bcrobes  Jakarta Indonesia"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Indra","family":"Wijaya","sequence":"additional","affiliation":[{"name":"Hasan Sadikin General Hospital  Bandung Indonesia"}],"role":[{"vocabulary":"crossref","role":"author"}]}],"member":"311","published-online":{"date-parts":[[2026,8,31]]},"reference":[{"key":"e_1_2_12_2_1","doi-asserted-by":"crossref","DOI":"10.3389\/fpubh.2025.1450625","article-title":"The Role of Public Health in Rare Diseases: Hemophilia as an Example","volume":"13","author":"El\u2010Sayed A. 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