National Hemophilia Foundation - for all bleeding disorders https://www.hemophilia.org/ en Important Changes Coming to Your State Medicaid or CHIP Program https://www.hemophilia.org/news/important-changes-coming-to-your-state-medicaid-or-chip-program <p><em>[This content was shared for public use by the Centers for Medicare &amp; Medicaid Services]</em></p> Thu, 02 Feb 2023 14:57:23 -0500 iostrin https://www.hemophilia.org/6528 WFH Gene Therapy Registry Goes Live https://www.hemophilia.org/news/wfh-gene-therapy-registry-goes-live <p><span><span><span><span><span><span>The World Federation of Hemophilia (WFH) has launched a new registry to help monitor the long-term safety and efficacy of hemophilia gene therapies in people with hemophilia across the globe. </span></span></span></span></span></span></p> Tue, 31 Jan 2023 10:01:25 -0500 jangarola https://www.hemophilia.org/6522 Sanofi Announces New Efanesoctocog alfa Data Published in the NEJM https://www.hemophilia.org/news/sanofi-announces-new-efanesoctocog-alfa-data-published-in-the-nejm <p><span><span><span><span><span><span>Sanofi recently announced newly published clinical trial data for efanesoctocog alfa, the company’s investigational recombinant factor VIII therapy (rFVIII) for the prevention of bleeding episodes in hemophilia A patients via once weekly prophylactic intravenous infusions.</span></span></span></span></span></span></p> Mon, 30 Jan 2023 14:47:22 -0500 jangarola https://www.hemophilia.org/6521 NHF Responds to FDA's Proposed Blood Donation Changes https://www.hemophilia.org/news/nhf-responds-to-fdas-proposed-blood-donation-changes <p class="x"><em><strong>FOR IMMEDIATE RELEASE</strong></em><br /> Contact: <a href="mailto:iostrin@hemophilia.org" rel=" noopener" target="_blank" title="Media Contact">Ilana Ostrin</a><br /> 212-328-3769<br /></p> Fri, 27 Jan 2023 16:30:26 -0500 iostrin https://www.hemophilia.org/6520 NHF & HFA Respond to New Blue Cross Blue Shield of Tennessee Formularies https://www.hemophilia.org/news/nhf-hfa-respond-to-new-blue-cross-blue-shield-of-tennessee-formularies <p><strong><em>FOR IMMEDIATE RELEASE</em><br /> Contact:</strong><br /><a href="mailto:iostrin@hemophilia.org" rel=" noopener" target="_blank" title="Media Contact">Ilana Ostrin</a><br /> 212-328-3769<br /></p> Fri, 20 Jan 2023 10:00:00 -0500 iostrin https://www.hemophilia.org/6490 New FIX Chromogenic Assay Launched by Precision BioLogic https://www.hemophilia.org/news/new-fix-chromogenic-assay-launched-by-precision-biologic <p><span><span><span><span><span><span>Precision BioLogic Inc., a company specializing in hemostasis diagnostics, recently announced that they have received the requisite clearance from the U.S. Food and Drug Administration (FDA) to launch and market their CRYO<i>check</i>™ Chromogenic Factor IX assay. </span></span></span></span></span></span></p> Wed, 18 Jan 2023 09:48:51 -0500 jangarola https://www.hemophilia.org/6482 Biomarin Provides Hemophilia A Clinical Development Program Updates https://www.hemophilia.org/news/biomarin-provides-hemophilia-a-clinical-development-program-updates <p class="body-style">BioMarin recently shared a community update on their Hemophilia A Clinical Development Program for valoctocogene roxaparvovec, the company’s investigational gene therapy currently under review by the U.S. Food and Drug Administration (FDA). It included a brief clinical trial overview with the following updates:</p> <ul><li> <p class="body-style">The Phase 1/2 study was initiated in 2015 and involved 15 individuals and two dose levels. Participants are now in long term follow-up post treatment with valoctocogene roxaparvovec.</p></li></ul> Tue, 10 Jan 2023 15:17:55 -0500 jangarola https://www.hemophilia.org/6476 Takeda Announces Trial Results on Investigational Therapy for Ultra Rare Blood Disorder https://www.hemophilia.org/news/takeda-announces-trial-results-on-investigational-therapy-for-ultra-rare-blood-disorder <p>Takeda recently announced favorable results from a phase 3 study of TAK-755, the company’s investigational enzyme replacement therapy for an ultra-rare blood disorder known as congenital thrombotic thrombocytopenic purpura (cTTP).</p> Tue, 10 Jan 2023 13:48:26 -0500 jangarola https://www.hemophilia.org/6475 NHF Announces 2023 Winter/Spring Wednesday Webinar Schedule https://www.hemophilia.org/news/nhf-announces-2023-winterspring-wednesday-webinar-schedule <p>FOR IMMEDIATE RELEASE<br /> Ilana Ostrin<br /> Senior Director of Public Relations and Communications<br /><a href="mailto:iostrin@hemophilia.org">iostrin@hemophilia.org</a><br /> 212-328-3769</p> <p class="text-align-center"> <br /><strong><em>NHF Announces 2023 Winter/Spring Wednesday Webinar Schedule</em><br /> The free series will continue in the new year with topics including gene editing experiments, innovative insurance cards, substance use, and more.</strong><br />  </p> Thu, 05 Jan 2023 10:25:47 -0500 cfellwock https://www.hemophilia.org/6474 Pfizer Announces Trial Updates for Investigational Hemophilia Gene Therapy https://www.hemophilia.org/news/pfizer-announces-trial-updates-for-investigational-hemophilia-gene-therapy <p><span><span><span><span><span><span>Pfizer recently announced positive top-line results from the phase 3 BENEGENE-2 clinical study, which is currently evaluating the investigational gene therapy fidanacogene elaparvovec for the treatment of adult males with moderately severe to severe hemophilia B.</span></span></span></span></span></span></p> Wed, 04 Jan 2023 12:41:38 -0500 jangarola https://www.hemophilia.org/6471