Beta thalassemia: Beta thalassemia is a blood disorder that reduces the production of hemoglobin. Hemoglobin is the iron-containing protein in red blood cells that carries oxygen to cells throughout the body. In people with beta thalassemia, low levels of hemoglobin lead to a lack of oxygen in many parts of the body. Contents: 1) Introduction In Southeast Asia alpha-thalassemia, beta-thalassemia, hemoglobin (Hb) E and Hb Constant Spring are prevalent. The gene frequencies of alpha-thalassemia reach % in Northern Thailand and Laos. beta-Thalassemia gene frequencies vary between 1 and 9%. Hb E is the hallmark of Southeast Asia attaining a frequency of % at the junction of Thailand, Laos, and Cambodia In hospital-based studies, alpha(+)-thalassemia has been found to protect against severe, life-threatening falciparum malaria. alpha(+)-Thalassemia does not seem to prevent infection or high parasite densities but rather limits progression to severe disease--in particular, severe malarial anemia. We assessed to what extent alpha(+)-thalassemia influences the association between mild,
Research Paper On Thalassemia - Words | Internet Public Library
What is Thalassemia? Thalassemia Research Studies By Lisa Du, Study Coordinator II Title Abstract Natural History of Iron Burden and Risk of Organ Injury as Assessed and Predicted by Non-Invasive Measurement Techniques Longitudinal assessment of whole body iron burden is essential for managing chelation and phlebotomy therapies and may be effective in predicting risk of organ injury.
Biomagnetic susceptibility measurement of liver iron concentration using SQUID technology. We will assess iron burden by biosusceptometry and serum ferritin at CHRCO and evaluate the clinical evidence of cardiac, hepatic, endocrine and orthopedic dysfuntion, and relate it to total iron burden as assessed by biosusceptometry and other non-invasive techniques.
Evaluate the Efficacy and Safety of RBCs Derived from Mirasol-treated Whole Blood Compared with Conventional RBCs in Patients Requiring Chronic Transfusion Support PRAISE Trial Thalassemia is the most transfused syndrome worldwide.
The risk of transmitting pathogens is reduced by pre-screening of blood donors and testing of the blood, papers research thalassemia. The Mirasol System offers a means to make transfusions significantly safer by targeting unscreened and undetected pathogens. This is a prospective, multi-center, randomized, crossover trial to evaluate the clinical effectiveness of RBCs derived from Mirasol-treated WB versus conventional RBCs in transfusion dependent thalassemia patients.
Towards the Development of a Noninvasive Prenatal Testing for Beta-Hemaglobinopathies The goal of this project is to show proof of concept for a non-invasive prenatal test NIPT for beta-hemoglobinopathies utilizing a novel DNA probe capture assay and next generation sequencing NGS. The final proof of principle for this NIPT assay requires blood samples from pregnant couples, confirmed to have mutations in the beta-globin gene, papers research thalassemia.
For this work we are collaborating with our Indian colleagues at the Postgraduate Institute of Medical Education and Research, papers research thalassemia, Chandigarh. A Multi-center, Randomized, Open-Label, Parallel-Group Study with LJPC for the Treatment of Myocardial Iron Overload in Adult Patients with Transfusion-Dependent Beta Thalassemia This is a study of hepcidin LJPC in adults with transfusion-dependent beta-thalassemia and cardiac iron overload. This is an open-label study in which half of the participants will receive weekly subcutaneous injections of hepcidin in addition to their regular treatment for 52 weeks.
The rest of the participants will receive regular standard of care for the first 26 weeks, and then will receive weekly hepcidin injections for the next 26 weeks in addition to their regular treatment.
This study is designed to enroll patients across multiple sites. BCHO plans to enroll up to 5 patients. Hepcidin is a substance produced by the liver to control the iron levels in plasma. Changing the amount of hepcidin allows control of iron released into the plasma from body stores and absorption of iron from food. As such, synthetic hepcidin may help in controlling iron excess. The aim of this protocol is to evaluate the effect of treatment with hepcidin on iron overload in the heart muscle.
The study will also evaluate if the use of hepcidin safe and tolerable, papers research thalassemia. A Phase 2, Open-Label, Multicenter Study to Determine the Efficacy, Safety, Pharmacokinetic, and Pharmacodynamics of AG in Adult Subjects with Non-Transfusion-Dependent Thalassemia Pending This is an open-label papers research thalassemia study in patients with non-transfusion-dependent thalassemia, including patient with beta thalassemia and hemoglobin H constant spring disease.
Approximately 17 patients will be enrolled at 3 sites in the United States, papers research thalassemia. BCHO will enroll approximately 8 patients.
Thalassemia is an inherited blood disease where the body cannot make enough hemoglobin. This produces anemia that can be severe enough to require blood transfusions for survival. Many patients do not require regular transfusions, but are at risk from the effects of papers research thalassemia anemia, papers research thalassemia.
The treatment options for such patients are very limited. In this study, we are evaluating whether the study drug, papers research thalassemia, AG, which may improve the energy metabolism of red blood cells, will lead to improved overall fitness and survival of blood cells. Papers research thalassemia study will include patients with either beta thalassemia intermedia, or hemoglobin H Constant Spring disease. The primary objectives of this trial are to measure the improvement in hemoglobin level in response to treatment with AG Secondary objectives are to evaluate the safety and pharmacokinetics or AG and to determine the effect of the AG on markers of hemolysis, erythropoeitic activity and iron metabolism.
A Phase 2 Study of PTG in Non-Transfusion Dependent NTD and Transfusion-Dependent TD ß- thalassemia Subjects with Chronic Anemia Pending This is an open-label study in which papers research thalassemia will receive weekly subcutaneous injections of PTG in addition to their regular standard of care for 16 weeks.
The study participants will receive the study drug at different dosage with the potential to titrate up in accordance with safety and efficacy evaluation. This study is designed to enroll 84 patients across multiple sites. BCHO plans to enroll up to 2 patients. Changing the amount of hepcidin with the hepcidin mimic allows control of iron released into the plasma from body stores and absorption of iron from food. Ineffective erythropoiesis is the papers research thalassemia of ß- thalassemia that elicits a number of compensatory mechanisms resulting in erythroid marrow expansion, extramedullary hematopoiesis, splenomegaly, and increased gastrointestinal iron absorption.
As such, a synthetic hepcidin mimic may result in iron redistribution in ß-thalassemia subjects with potentially beneficial effects on erythropoiesis and consequently improvements in chronic anemia. Video: Gene Therapy -- The time is now -- Nick Leschly, BluebirdBio. Gene Therapy Fact Sheet. Northern California Comprehensive Thalassemia Center.
UCSF Benioff Children's Hospital Oakland. Longitudinal assessment of whole body iron burden is essential for managing chelation and phlebotomy therapies and may be effective in predicting risk of organ injury. Evaluate the Efficacy and Safety of RBCs Derived from Mirasol-treated Whole Blood Compared with Conventional RBCs in Patients Requiring Papers research thalassemia Transfusion Support PRAISE Trial.
Thalassemia is the most transfused syndrome worldwide. Towards the Development of a Noninvasive Prenatal Testing for Beta-Hemaglobinopathies. The goal of this project is to show proof of concept for a non-invasive prenatal test NIPT for beta-hemoglobinopathies utilizing a novel DNA probe papers research thalassemia assay and next generation sequencing NGS. This gene therapy study is a single-arm, multi-site, single dose, phase 3 study to evaluate the safety and efficacy of autologous hematopoietic stem cell transplantation HSCT using LentiGlobin® BB Drug Product in patients with ß-thalassemia major.
This gene therapy study is a single-arm, papers research thalassemia, multi-site, single dose, phase 3 study to evaluate the safety and efficacy of autologous hematopoietic stem cell transplantation HSCT using LentiGlobin® BB Drug Product in patients with transfusion dependent ß-thalassemia. A Multi-center, Randomized, Open-Label, Parallel-Group Study with LJPC for the Treatment of Myocardial Iron Overload in Adult Patients with Transfusion-Dependent Beta Thalassemia. This is a study of hepcidin LJPC in adults with transfusion-dependent beta-thalassemia and cardiac iron overload.
Papers research thalassemia Phase 2, Open-Label, Multicenter Study to Determine the Efficacy, Safety, Pharmacokinetic, and Pharmacodynamics of AG in Adult Subjects with Non-Transfusion-Dependent Thalassemia Pending. This is an open-label multi-center study in patients with non-transfusion-dependent thalassemia, including patient with beta thalassemia and hemoglobin H constant spring disease.
A Phase 2 Study of PTG in Non-Transfusion Dependent NTD and Transfusion-Dependent TD ß- thalassemia Subjects with Chronic Anemia Pending. This is an open-label study in which participants will receive weekly subcutaneous injections of PTG in addition to their regular standard of care for 16 weeks, papers research thalassemia.
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Beta thalassemia: Beta thalassemia is a blood disorder that reduces the production of hemoglobin. Hemoglobin is the iron-containing protein in red blood cells that carries oxygen to cells throughout the body. In people with beta thalassemia, low levels of hemoglobin lead to a lack of oxygen in many parts of the body. Contents: 1) Introduction Results In a total of cases ( thalassemia major and 79 intermedia), were males (%) and females (%); mean age 22 years, range years. Alloimmunization was detected in 50(%) patients, including 37(74%) patients with one alloantibody, 8(16%) with two antibodies, 4(8%) patients with unknown antibodies and one patient (2%) with autoantibody Review Article. ISSN No. Abstract. Thalassemia’s ar e genetic disorders inherited from a person’s parents. Thalassemia’s are. prevalent worldwide with 25, deaths in Estimated Reading Time: 5 mins
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