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Showing 1 to 20 of 33 for “"hemophilia"”.
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GLOBAL COAGULATION ASSAYS AND HEMOPHILIA ARTHROPATHY SCORES FOR MONITORING EMICIZUMAB PROPHYLAXIS IN PATIENTS WITH HEMOPHILIA A
Introduzione: Le terapie non sostitutive stanno emergendo come valido trattamento nei pazienti affetti da emofilia. Tuttavia, nonostante i risultati promettenti, non esiste una tecnica di monitoraggio. La prima terapia non sostitutiva approvata per il trattamento dei pazienti affetti da emofilia A …
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Quality of life: Psychological, family, and social correlates in children with hemophilia.
… family, and social correlates in children with hemophilia.
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Improving the Safety and Efficacy of AAV Gene Therapy for Hemophilia A
Gene therapy for hemophilia A (HA) remains an unrealized therapeutic goal due to outstanding questions regarding safety and long-term efficacy after adeno-associated viral (AAV) vector delivery of the F8 gene to hepatocytes. To address questions of safety, we treated privately owned dogs with a …
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ENHANCEMENT OF hFVIII ACTIVITY THROUGH LC MODIFICATIONS FOR GENE THERAPY OF HEMOPHILIA A
Gene therapy for Hemophilia A (HA) using the recombinant Adeno-associated virus (rAAV) offers an alternative to classic treatment, which consists of FVIII protein infusions. However, due to limitations associated with rAAV and the FVIII protein itself, the end result is a transgene expression below …
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PIECING TOGETHER THE PUZZLE OF TREATING HEMOPHILIA A UTILIZING AAV BASED GENE THERAPY
… mutation or deletion. Disorders range from Hemophilia, Cystic fibrosis, and Duchenne muscular dystrophy to diseases like Cancer and Huntington’s Disease. While these diseases may seem vastly different, they all are due to issues in the genetic makeup of the patient. Gene therapy treats or …
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Measuring And Understanding Health-Related Quality Of Life Among Adult Patients With Hemophilia
… health-related quality (HRQOL) among adults with hemophilia in the United States. Methods: This cross-sectional study was conducted using both a web-based and paper-based self-administered survey. Adults with hemophilia were recruited using an online panel, a Facebook community of hemophilia …
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Evaluation of the determinants and characterization of bleeding in hemophilia A and B carriers
Hemophilia A and B are bleeding disorders caused by a deficiency in coagulation factor VIII (FVIII) or coagulation factor IX (FIX), respectively. Due to the X-linked inheritance pattern, males are affected with the disease, while females are almost exclusively carriers of hemophilia. Although …
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Stimulation Through Tlr4 Increases Fviii Inhibitor Formation In A Mouse Model of Hemophilia A
<p>Hemophilia A is a clotting disorder caused by functional factor VIII (FVIII) deficiency. About 25% of patients treated with therapeutic recombinant FVIII develop antibodies (inhibitors) that render subsequent FVIII treatments ineffective. The immune mechanisms of inhibitor formation are not …
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Infusing Factor Viii-Expressing Platelets Or Megakaryocytes As A Novel Therapeutic Strategy For Hemophilia A
… most common inherited form of severe bleeding, hemophilia A, a deficiency of functional coagulation factor VIII. Patients with severe hemophilia A suffer from recurrent bleeding with significant morbidity and mortality with 20-30% of these patients developing antibodies to infused Factor (F) …
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An Assessment of Knowledge and Attitudes of Genetic Counseling Services In U.S. Htcs
<p>Hemophilia is a hereditary bleeding disorder which requires lifelong specialized care. A network of Hemophilia Treatment Centers (HTCs) exists to meet the medical needs of patients affected by hemophilia. Genetic counseling services are an integral part of the HTC model of care; however, many …
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In Vivo Genome Editing: Proof of Concept in Neonatal and Adult Mouse Liver
… gene delivery for monogenic diseases, including hemophilia. Major limitations of this approach are the inability to persist in dividing cells and the restrictive packaging capacity of AAV. Gene targeting, the ability to make site-directed changes to the genome, has been a powerful tool for …
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Characterization of the Recombinant Human Factor VIII Expressed in the Milk of Transgenic Swine
… therapeutic applications for the treatment of Hemophilia A. Its deficiency, either qualitative or quantitative, results in Hemophilia A, a disorder affecting approximately 1 in 10,000 males. Currently, FVIII replacement therapy uses FVIII derived from plasma or cell culture. The current cost of …
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AUTOMATION MODEL FOR pH AND CONDUCTIVITY ADJUSTMENT FOR A CATION EXCHANGE COLUMN ELUATE BUFFER
… facility manufacturess Kogenate - a drug for Hemophilia. This project was carried out at one of the intermediate purification steps of Kogenate, where the pH and conductivity of a buffer solution added to a chromatographic column's eluate has to be critically adjusted. Currently this process …
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Characterization of Post-translational Modifications and Resulting Structure/Function Relationships of Recombinant Human Factor IX Produced in the Milk of Transgenic Pigs
Hemophilia B is a debilitating and life-threatening disorder caused by a deficiency in or dysfunction of factor IX (FIX), a complex plasma glycoprotein required for the formation and maintenance of blood clots. Treatment of hemophilia B involves infusion of replacement FIX currently derived from …
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FVIII Immunity : early events and tolerance mechanisms to FVIII
Among the complications of current treatments for hemophilia A, the development of anti-FVIII antibodies including “FVIII inhibitors” remains the major clinical problem in treating hemophiliacs. Factor VIII inhibitors work through neutralizing the coagulation cofactor activity of the infused FVIII …
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MITIGATING DATA SCARCITY CHALLENGES IN MEDICAL IMAGING ANALYSIS:ADVANCED LEARNING APPROACHES WITH EMPHASIS ON HEMOPHILIC ULTRASOUND IMAGES
Medical imaging plays a crucial role in hemophilia research and clinical practice, particularly in assessing joint health and bleeding events. Ultrasound (US) imaging is a fundamental tool in the diagnostic process and is currently used to identify when the joint recess is filled with synovial …
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Novel carriers for oral delivery of hemophiliac factor IX
Current treatments for hemophilia B, a hereditary bleeding disorder characterized by the deficiency of coagulation factor IX (FIX), rely on injection-based administration that cause pain and discomfort, leading to noncompliance and risk of subsequent bleeding episodes. A non-invasive protein …
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Exploring Camper Perspectives on Health Care Transition Programming Offered Through Medical Specialty Camps
… valued by campers with sickle cell disease or hemophilia. Nine pre-transition youth (16- 22 years) and four post-transition adults (22-36 years) who attended medical specialty camps run concurrently in southeastern United States participated in semi-structured interviews. Interviews were …
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Cavitation in Pharmaceutical Manufacturing and Shipping
… proteins are used to successfully treat hemophilia, Crohn's Disease, diabetes, and cancer. Recent product recalls have occurred because of sub-visible particle formation resulting from the inherent instability of proteins. It has been suggested that particle formation is associated with …
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Adeno-associated virus type 2 as vector for human gene therapy: Characterization of virus-host interactions
… of various diseases (e.g. cystic fibrosis, hemophilia B, cancer). Limitations, however, still exist and require further improvement. The study presented here addresses two major problems that hamper a widespread use of AAV in human gene therapy: First, the loss of site-specific integration …
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