BioMarin Submits Biologics License Application to U.S. Food and Drug Administration for Valoctocogene Roxaparvovec to Treat Hemophilia A

SAN RAFAEL, Calif., Dec. 23, 2019 /PRNewswire/ -- BioMarin Pharmaceutical Inc. (NASDAQ: BMRN) announced today that the company submitted a Biologics License Application (BLA) to the U.S. Food and Drug Administration (FDA) for its...
Source: Drugs.com - New Drug Applications - Category: Drugs & Pharmacology Source Type: news

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INTRODUCTION: Sports participation in children with haemophilia is generally considered to be associated with increased injury risk, which is generally considered highest in severe haemophilia. AIM: To assess sports participation according to age a...
Source: SafetyLit - Category: International Medicine & Public Health Tags: Age: Adolescents Source Type: news
LOUISVILLE, Ky., April 6, 2020 /PRNewswire/ -- HEMA Biologics, LLC, ( " HEMA Biologics " ) today announced that the U.S. Food and Drug Administration (FDA) approved Sevenfact® [coagulation factor VIIa (recombinant)-jncw] as the first new bypassing...
Source: Drugs.com - New Drug Approvals - Category: Drugs & Pharmacology Source Type: news
First new bypassing agent approved for the treatment and control of bleeding episodes in hemophilia A and B patients with inhibitors in over 2 decades Median number of infusions required to achieve bleeding control in the first 12 hours was 1 (225 mcg/k... Biopharmaceuticals, FDA HEMA Biologics, SEVENFACT, coagulation factor VIIa, hemophilia
Source: HSMN NewsFeed - Category: Pharmaceuticals Source Type: news
THE MANAGEMENT of inherited or acquired coagulation disorders can be challenging during cardiac surgery with extracorporeal circulation. Although guidelines recommend preoperative supplementation of factor XI (FXI) with severe hemophilia C, there are certain risks involved with this strategy with the application of cardiac bypass.
Source: Journal of Cardiothoracic and Vascular Anesthesia - Category: Anesthesiology Authors: Tags: Case Report Source Type: research
Abstract Emicizumab is a humanized anti-FIX/FX (factor IXa/X) bispecific monoclonal antibody that mimics FVIIIa (activated factor VIII) cofactor function. The hemostatic efficacy of emicizumab has been confirmed in clinical studies of patients with hemophilia A, irrespective of the presence of FVIII inhibitors. Emicizumab differs in some properties from FVIIIa molecule. Emicizumab requires no activation by thrombin and is not inactivated by activated protein C, but emicizumab-mediated coagulation is regulatable and maintains hemostasis. A small amount of FIXa (activated factor IX) is required to initiate emicizuma...
Source: Arteriosclerosis, Thrombosis and Vascular Biology - Category: Cardiology Authors: Tags: Arterioscler Thromb Vasc Biol Source Type: research
Hemophilia and von Willebrand disease (VWD) are the most common congenital coagulation factor deficiencies. Patients with these disorders who experience bleeding complications are often initially managed in the emergency department (ED).
Source: The Journal of Emergency Medicine - Category: Emergency Medicine Authors: Tags: Clinical Reviews Source Type: research
Acquired hemophilia A (AHA) is a rare autoimmune disease due to acquired IgG1-4 autoantibodies against coagulation factor VIII (FVIII). This causes patients, who have negative family and personal histories, to present with a sudden onset of heavy bleeding.1 –3 Although rare, the presence of antibodies against factors V and IX is possible.4
Source: Pediatrics and Neonatology - Category: Perinatology & Neonatology Authors: Tags: Letter to the Editor Source Type: research
April 01, 2020 -- The U.S. Food and Drug Administration today approved Sevenfact [coagulation factor VIIa (recombinant)-jncw] for the treatment and control of bleeding episodes occurring in adults and adolescents 12 years of age and older with...
Source: Drugs.com - New Drug Approvals - Category: Drugs & Pharmacology Source Type: news
‚ÄčThe wrist is not commonly aspirated in the emergency department, but emergent arthrocentesis may be indicated for extreme or concerning cases, and tapping the wrist to determine the underlying pathology or relieve pain may be of great value. The synovial fluid from the joint space can be analyzed for crystals, infection, and blood. This information may help determine the overall plan and aid in decision-making and consultation. The ultimate treatment plan may include admission, intravenous antibiotics, multiple aspirations, and even surgical washout.A swollen, painful wrist that is hot to the touch is concerning for sep...
Source: The Procedural Pause - Category: Emergency Medicine Tags: Blog Posts Source Type: blogs
The objective was to review the characteristics and the current understanding of mechanisms contributing to assay discrepancy in DHA. METHODS: Characteristics of the DHA patients treated were examined by retrospective chart review. In addition, a literature review was performed to determine the current understanding of DHA. RESULTS: Three cases of DHA were diagnosed based on bleeding phenotype: 2 cases represented missed diagnoses of HA, and 1 represented misclassification of hemophilia severity. The revised diagnosis and classification of hemophilia directly affected clinical management. Review of the literature...
Source: American Journal of Clinical Pathology - Category: Pathology Authors: Tags: Am J Clin Pathol Source Type: research
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