This page keeps track of new/upcoming treatment options for hemophilia and bleeding disorders. What’s in development? What’s promising? What’s available soon? Find out below…
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News
- Newly Approved Drug Being Called ‘Game Changer’ For People Who Suffer From Hemophilia Sep 4, 2019
- UniQure stays a step ahead in hemophilia B Sep 3, 2019
- Pediatric hematologists introduce a novel way to treat pediatric hemophilia A patients Aug 29, 2019
- Recent advances in thrombin-targeting therapeutic strategies that aim to prevent bleeding in patients with hemophilia and other rare bleeding disorders Jun 7, 2019
- BioMarin says data shows hemophilia gene therapy effects could wane May 28, 2019
- Biomarin to seek approval for hemophilia gene therapy, but durability questions persist May 28, 2019
- Gene Therapy: Paving New Roads in the Treatment of Hemophilia May 16, 2019
- Potential Gene Therapy Safely Restores FVIII Clotting Factor in Animals and Human Cells, ASC Therapeutics Reports May 3, 2019
- uniQure Announces New Preclinical Data in Hemophilia A [AMT-180] in Oral Presentations at the 22nd ASGCT Annual Meeting May 2, 2019
- Sigilon Therapeutics Delivers Oral Presentation on SIG-001 for Hemophilia A at 2019 ASGCT Meeting May 1, 2019
- Applied StemCell Therapeutics Cures Hemophilia A in Mice Apr 25, 2019
- “A number of biopharma companies are making progress in developing [hemophilia] treatments, therapies, and potentially even cures” Apr 17, 2019
- Gene-based factor VIIa prevents bleeding episodes in animals with hemophilia Apr 15, 2019
Research
- Hemophilia A ameliorated in mice by CRISPR-based in vivo genome editing of human Factor VIII Nov 14, 2019
- Mesenchymal stem cells as carrier of the therapeutic agent in the gene therapy of blood disorders Nov 6, 2019
- Tissue Factor-Negative Cell-Derived Microparticles Play a Distinctive Role in Hemostasis Jun 13, 2019
- Limitations of prophylactic treatment in patients with hemophilia May 30, 2019
- The central role of thrombin in bleeding disorders May 22, 2019
- Gene Therapy: Paving New Roads in the Treatment of Hemophilia May 16, 2019
- Current and Emerging Biologics for the Treatment of Hemophilia Apr 30, 2019
- Efficacy, safety, and pharmacokinetics of emicizumab prophylaxis given every 4 weeks in people with haemophilia A Apr 16, 2019
Clinical Trials
- Safety and Efficacy of Nonacog Beta Pegol (N9-GP) in Previously Untreated Patients With Haemophilia B Jun 6, 2019
- Fitusiran Prophylaxis in Male Pediatric Subjects Aged 1 to Less Than 12 Years With Hemophilia A or B Jun 4, 2019
- Lentiviral FVIII Gene Therapy for Hemophilia A [YUVA-GT-F801] May 23, 2019
- A Trial Evaluating Efficacy and Safety of Prophylactic Administration of Concizumab in Patients With Severe Haemophilia A Without Inhibitors May 22, 2019
- A Trial Evaluating the Efficacy and Safety of Prophylactic Administration of Concizumab in Haemophilia A and B Patients With Inhibitors May 22, 2019
- A Study of Fitusiran (ALN-AT3SC) in Severe Hemophilia A and B Patients Without Inhibitors May 15, 2019
- Clinical Trial Comparing a Biosimilar Eptacog Alfa With Novoseven, in Patients With Hemophilia With Inhibitors May 2, 2019
- Study to evaluate safety, immunogenicity and hemostatic efficacy of PEGylated Recombinant Factor VIII (BAX 855) in previously untreated patients with severe hemophilia A Apr 25, 2019
- Long-term Safety and Efficacy Study of SPK-9001 in Individuals With Hemophilia B Apr 24, 2019
- Gene Therapy Trial for Platelet Derived Factor VIII Production in Hemophilia A Apr 23, 2019
