{"id":3255,"date":"2021-12-08T14:22:00","date_gmt":"2021-12-08T14:22:00","guid":{"rendered":"https:\/\/fusfoundation.org\/?post_type=sw_disease_condition&#038;p=3255"},"modified":"2025-06-05T10:57:23","modified_gmt":"2025-06-05T14:57:23","slug":"hemophilia","status":"publish","type":"sw_disease_condition","link":"https:\/\/www.fusfoundation.org\/diseases-and-conditions\/hemophilia\/","title":{"rendered":"Hemophilia"},"content":{"rendered":"\n<h2 class=\"wp-block-heading\" id=\"h-focused-ultrasound-therapy\">Focused Ultrasound Therapy<\/h2>\n\n\n\n<p>Focused ultrasound is a noninvasive, therapeutic technology with the potential to improve the quality of life and decrease the cost of care for patients with hemophilia B, also known as Christmas Disease or Factor IX Deficiency. Similar work is being done with Hemophilia A. This novel technology focuses beams of ultrasound energy precisely and accurately on targets in the liver.<\/p>\n\n\n\n<p><strong>How it Works<\/strong><br>Where the beams converge, focused ultrasound enables the release of gene-expressing vectors (in Hemophilia B) and non-viral genes (Hemophilia A), without damaging surrounding normal tissue. This preclinical work will be important in the development of future human trials.<\/p>\n\n\n\n<p><strong>Advantages<\/strong><br>For certain patients, focused ultrasound could provide a noninvasive alternative treatment with less risk of complications \u2013 such as surgical wound healing or infection \u2013 at a lower cost. It can reach the desired target without damaging surrounding tissue and is repeatable, if necessary.<\/p>\n\n\n\n<h2 class=\"wp-block-heading\" id=\"h-clinical-trials\">Clinical Trials<\/h2>\n\n\n\n<p>At the present time, there are no clinical trials recruiting patients for focused ultrasound enhancement of gene-expression vectors to treat Hemophilia.<\/p>\n\n\n\n<h2 class=\"wp-block-heading\" id=\"h-regulatory-approval-and-reimbursement\">Regulatory Approval and Reimbursement<\/h2>\n\n\n\n<p>Focused ultrasound treatment for enhancing the gene-expression treatment for Hemophilia is not yet approved by regulatory bodies or covered by medical insurance companies.<\/p>\n\n\n\n<p>The Foundation updates these pages regularly, but with the increasing number of clinical trials, we want to be sure that our audience has the latest information available. Therefore, we also added the website search information for the above trials. If you click <a href=\"https:\/\/clinicaltrials.gov\/search?cond=Hemophilia&amp;term=focused%20ultrasound&amp;aggFilters=status:not%20rec\" target=\"_blank\" rel=\"noreferrer noopener\"><strong>here<\/strong><\/a>, it will take you to the latest information available from <a href=\"https:\/\/www.clinicaltrials.gov\/\" target=\"_blank\" rel=\"noreferrer noopener\"><strong>https:\/\/www.clinicaltrials.gov\/<\/strong><\/a>.\u00a0<\/p>\n\n\n\n<h2 class=\"wp-block-heading\" id=\"h-notable-papers\">Notable Papers<\/h2>\n\n\n\n<p>Lo WS, Sheen JM, Chen YC, Wu KT, Wang LY, Lau YC, Hsiao CC, Ko JY. <a aria-label=\"The Application of Focused Medium-Energy Extracorporeal Shockwave Therapy in Hemophilic A Arthropathy.  (opens in a new tab)\" class=\"ek-link\" href=\"https:\/\/pubmed.ncbi.nlm.nih.gov\/35206968\/\" target=\"_blank\" rel=\"noreferrer noopener\"><strong>The Application of Focused Medium-Energy Extracorporeal Shockwave Therapy in Hemophilic A Arthropathy<\/strong><\/a>. Healthcare (Basel). 2022 Feb 11;10(2):352. doi: 10.3390\/healthcare10020352.<\/p>\n\n\n\n<p>Tran DM, Zhang F, Morrison KP, Loeb KR, Harrang J, Kajimoto M, Chavez F, Wu L, Miao CH.&nbsp;<a href=\"https:\/\/www.ncbi.nlm.nih.gov\/pubmed\/31497618\" target=\"_blank\" rel=\"noreferrer noopener\"><strong>Transcutaneous Ultrasound-Mediated Nonviral Gene Delivery to the Liver in a Porcine Model<\/strong><\/a>. Mol Ther Methods Clin Dev Jul 26 2019; 14:275-284. doi: 10.1016\/j.omtm.2019.07.005.<\/p>\n\n\n\n<p><a href=\"https:\/\/www.youtube.com\/watch?v=EO7DLYvJrkI\" target=\"_blank\" rel=\"noreferrer noopener\"><strong>High Intensity Focused Ultrasound for Hepatic Gene Transfer<\/strong><\/a>. Presentation by Cynthia Anderson, PhD. 2018<\/p>\n\n\n\n<p><em><strong><a href=\"https:\/\/pubmed.ncbi.nlm.nih.gov\/?term=(%22focused%20ultrasound%22%20OR%20HIFU)%20and%20%22platelet%22&amp;sort=date\" target=\"_blank\" aria-label=\"Click here (opens in a new tab)\" rel=\"noreferrer noopener\" class=\"ek-link\">Click here<\/a>&nbsp;for additional references from PubMed.<\/strong><\/em><\/p>\n","protected":false},"template":"","meta":{"_acf_changed":false,"_searchwp_excluded":"","_editorskit_title_hidden":false,"_editorskit_reading_time":1,"_editorskit_is_block_options_detached":false,"_editorskit_block_options_position":"{}","episode_type":"","audio_file":"","podmotor_file_id":"","podmotor_episode_id":"","cover_image":"","cover_image_id":"","duration":"","filesize":"","filesize_raw":"","date_recorded":"","explicit":"","block":"","itunes_episode_number":"","itunes_title":"","itunes_season_number":"","itunes_episode_type":"","_vp_format_video_url":"","_vp_image_focal_point":[],"footnotes":""},"sw_disease_condition_cat":[29],"sw_disease_condition_stage":[14],"class_list":["post-3255","sw_disease_condition","type-sw_disease_condition","status-publish","hentry","sw_disease_condition_cat-miscellaneous"],"acf":[],"yoast_head":"<!-- This site is optimized with the Yoast SEO Premium plugin v27.2 (Yoast SEO v27.2) - 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