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Chugai Pharma Emicizumab Receives Priority Review Designation Hemophilia PubMed articles on BioPortfolio. Our PubMed references draw on over 21 million records from the medical literature. Here you can see the latest Chugai Pharma Emicizumab Receives Priority Review Designation Hemophilia articles that have been published worldwide.
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Emicizumab is a bispecific monoclonal antibody that bridges activated factor IX and factor X to replace the function of missing activated factor VIII, thereby restoring hemostasis. In a phase 3, multicenter trial, we investigated its use as prophylaxis in persons who have hemophilia A without factor VIII inhibitors.
Activated protein C (APC) inactivates activated factor V (FVa) and moderates FVIIIa by restricting FV cofactor function. Emicizumab is a humanized anti-FIXa/FX bispecific monoclonal antibody that mimicks FVIIIa cofactor function. In recent clinical trials in haemophilia A patients, once-weekly subcutaneous administration of emicizumab was remarkably effective in preventing bleeding events, but the mechanisms controlling the regulation of emicizumab-mediated haemostasis remain to be explored. We investigated...
Clinical coagulation assays are an integral part of diagnosing and managing patients with hemophilia; however, in this new era of bioengineered factor products and non-factor therapeutics, problems have arisen with use of traditional coagulation tests for the quantification of several of these new products. Discussion over the use of one-stage clotting assays versus chromogenic substrate assays for clinical decision making and potency labeling has been ongoing for many years. Emerging factor concentrates ha...
Previous retrospective claims database analyses reported increased prevalence and earlier onset of cardiovascular comorbidities in patients with versus without hemophilia A. A comprehensive chart review was designed to further investigate previous findings.
The concept of joint microbleeding in hemophilia patients was first proposed over 10 years ago. This was based on unexpected abnormalities found in medical imaging studies of asymptomatic joints. Since then, there have been no published studies confirming the presence of joint microbleeds. This critique will review the evidence for and against joint microbleeding in hemophilia patients and the potential implications. This article is protected by copyright. All rights reserved.
Prophylactic injections of factor VIII reduce the incidence of bleeds and slow the development of joint damage in people with hemophilia. The aim of this study was to identify optimal person-specific prophylaxis regimens for children with hemophilia A.
There is a great interest from global companies who are developing biosimilars to pursue interchangeability designation for commercialization of their products in the US. An interchangeability designation will not only allow the substitutability at the pharmacy level without the intervention of a health care provider but the first sponsor who is able to garner interchangeability designation will also receive 12 months of marketing exclusivity. This paper will highlight our current understanding of FDA expec...
Hemophilia B (HB) is an X-linked bleeding disorder caused by deficiency of factor IX (FIX). Patients with the severe form (FIX
Health-related quality of life (HRQoL) is impaired in patients with hemophilia, however the impact in mild/moderate hemophilia B and affected women is not well characterized.
The immunogenicity of therapeutic factor VIII (FVIII) in patients with hemophilia A has been puzzling scientific and clinical communities for more than 3 decades. Indeed, the development of inhibitory antibodies to FVIII remains a major clinical challenge and is associated with enormous societal costs. Thus, the reasons for which a presumably innocuous, short-lived, intravenously administered glycoprotein triggers such a deleterious, long-lasting neutralizing immune response is an enigma. This review does n...
The term Magnet hospital is an official designation ascribed by the American Nurses Credentialing Center for hospitals that meet specific criteria indicating they have a "magnetic work environment" for nurses. The objective of the Magnet designation is to encourage hospitals to design work in such a way as to attract and retain high-quality nurses and thus improve the quality of patient care. Empirical research has demonstrated that hospitals who earn a Magnet designation appear to have nurses who are more ...
To assess the reliability and validity of 6 patient-reported outcomes (PRO) instruments for evaluating health-related quality of life in adults with mild-severe hemophilia B and caregivers of children with hemophilia B, including affected women/girls.
Therapeutic advances over the past 30 years have led to longer life expectancy and improved quality of life (QOL) for persons with hemophilia. Access to innovative therapy may be compromised if treatment decisions are driven solely by cost. New strategies are needed to assess true therapeutic values, along with financial cost, as physicians, policymakers, payers and manufacturers work together to improve patient care.
Eradication of factor VIII (FVIII) specific neutralizing antibodies (also known as inhibitors) by the traditional method of immune tolerance induction (ITI) is costly and unsuccessful in one out of three patients. Furthermore, effective inhibitor prevention strategies are presently lacking. An overview is given in this narrative review of antidrug antibody prevention or eradication strategies that have been used in disorders beyond hemophilia A, with the aim of analyzing what we can learn from these strateg...
Case report of a 74-year-old male with acquired hemophilia A, whose etiology was not found, confirmed by hemocoagulation examinations, with clinical manifestation of protracted macroscopic hematuria, spontaneous skin and intramuscular bleedings. Treatment comprised conservative therapy administration of hemostyptics, red blood cells transfusions application of recombinant human factor VII (rFVII) and immunosuppression. Finally, patient died due to nosocomial infection caused by multidrug-resistant pulmonary...
Congress created the tropical disease priority review voucher program to stimulate new drug development for tropical diseases. An analysis of the pharmaceutical pipeline indicates that the development of drugs for these tropical diseases has increased. However, the effects of the program are not uniform across all diseases, as malaria and tuberculosis have seen significant new drug development, while other diseases have not.
Patients with hemophilia A are defined as "severe" if they present
Current research suggests that while patients are becoming more engaged across the health delivery spectrum, this involvement occurs most often at the pre-preparation stage to identify 'high-level' priorities in health ecosystem priority setting, and at the preparation phase for health research.
In the second decade of the third millennium there have been dramatic developments pertaining to the availability of highly innovative drugs for hemophilia care, notwithstanding a satisfactory previous scenario.
According to the Treaty on the Eurasian Economic Union, a common pharmaceutical market was supposed to become operational on January 1, 2016. Nevertheless, the responsible parties did not meet the deadline. Although a majority of the draft guidelines regulating this process were already known in 2014 and 2015, the anticipated process itself caused anxiety among representatives of the foreign pharma industry, who tried to prepare for coming challenges. Among other possible issues, the ambiguity of transition...
Hemophilia B is an ideal target for gene- and cell-based therapies because of its monogenic nature and broad therapeutic index. Here, we demonstrate the use of cell therapy as a potential long-term cure for hemophilia B in our FIX-deficient mouse model. We show that transplanted, cryopreserved, cadaveric human hepatocytes remain functional for more than a year and secrete FIX at therapeutic levels. Hepatocytes from different sources (companies and donors) perform comparably in curing the bleeding defect. We...
The binary designation 'Symbiodinium thermophilum' was invalid due to the absence of an illustration as required by Article 44.2 of the ICN. Herein, it is validated. This species is the most common symbiont in reef corals in the southern Persian/Arabian Gulf (PAG), the world's hottest body of water sustaining reef coral growth. This article is protected by copyright. All rights reserved.
A patient's perception of hospital or provider quality can have far-reaching effects, as it can impact reimbursement, patient selection of a surgeon, and healthcare competition. A variety of organizations offer quality designations for orthopedic surgery and its subspecialties. Our goal is to compare total joint arthroplasty (TJA) quality designation methodology across key quality rating organizations. One researcher conducted an initial Google search to determine organizations providing quality designation...